<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03304054</url>
  </required_header>
  <id_info>
    <org_study_id>MSK-002</org_study_id>
    <nct_id>NCT03304054</nct_id>
  </id_info>
  <brief_title>Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG</brief_title>
  <official_title>A Randomized, Placebo-control, Parallel Group Study to Evaluate the Effect of Amifampridine Phosphate in Patients With MuSK Antibody Positive Myasthenia Gravis, and a Sample of AChR Antibody Positive Myasthenia Gravis Patients</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Catalyst Pharmaceuticals, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Catalyst Pharmaceuticals, Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Efficacy and safety of amifampridine phosphate in improving the activities of daily living&#xD;
      for patients with antibody positive MuSK myasthenia gravis.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Randomized, double-blind, placebo-controlled, parallel group study is designed to evaluate&#xD;
      the safety, tolerability and efficacy of amifampridine phosphate in patients with MuSK-MG. In&#xD;
      addition, a sample of AChR-MG patients will be assess for efficacy and safety of&#xD;
      amifampridine phosphate. Planned duration of participation for each patient is at least 38&#xD;
      days, excluding the screening period. Eligible patients will be titrated to an efficacious&#xD;
      dose of amifampridine phosphate and those who demonstrate improvement will be randomized to&#xD;
      either placebo or amifampridine, in a double-blind fashion, for 10 days.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">April 18, 2018</start_date>
  <completion_date type="Actual">March 15, 2020</completion_date>
  <primary_completion_date type="Actual">January 31, 2020</primary_completion_date>
  <phase>Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results</measure>
    <time_frame>Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time point at which a patient discontinued treatment early).</time_frame>
    <description>Myasthenia Gravis-Activities of Daily Living (MG-ADL) is a self-report scale to assess the patient's MG symptoms and functional performance of activities of daily living. The eight items are scored on a scale of 0-3 with 3 representing the most severe symptoms or impaired performance and 0 representing no symptoms or impaired performance. Each item was assessed by the patient at the last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total MG-ADL score was calculated as the sum of each item score, with a maximum score of 24 (most severe symptoms/impairment) and minimum score of 0 (least severe symptoms/impairment). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnos</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Quantitative Myasthenia Gravis (QMG) Total Score Summary Statistics by Time Point and MG Type: Wilcoxon-Mann-Whitney Rank Sum Test Results</measure>
    <time_frame>Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time at which a patient discontinued treatment early).</time_frame>
    <description>Quantitative Myasthenia Gravis (QMG) assesses the patient's general body strength and fatigability. Each test item is scored on a scale of 0-3 with 3 representing the most severe symptom results and 0 representing no symptom results. Each item was assessed by the patient at Screening, the first (Day 1) and last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total QMG score was calculated as the sum of each item score, with a maximum score of 39 (most severe symptoms) and minimum score of 0 (least severe symptoms). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnosed with MuSK-MG treated with amifampridine and placebo was conducted.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">93</enrollment>
  <condition>Myasthenia Gravis, Generalized</condition>
  <arm_group>
    <arm_group_label>amifamapridine phosphate tablets</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>placebo tablets</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Amifampridine Phosphate</intervention_name>
    <description>tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day</description>
    <arm_group_label>amifamapridine phosphate tablets</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo Oral Tablet</intervention_name>
    <description>tablets matching amifampridine phosphate, 3 to 4 times a day</description>
    <arm_group_label>placebo tablets</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Willing and able to provide written informed consent after the nature of the study has&#xD;
             been explained and before the start of any research-related procedures.&#xD;
&#xD;
          2. Male or female ≥18 years of age.&#xD;
&#xD;
          3. Positive serologic test for anti-MuSK antibodies or anti-AChR antibodies as confirmed&#xD;
             at Screening or by previous antibody test, with report available.&#xD;
&#xD;
          4. Confirmatory EMG or EMG report.&#xD;
&#xD;
          5. Myasthenia Gravis Foundation of America (MGFA) Class II to IV at Screening.&#xD;
&#xD;
          6. MG-ADL score of ≥6 at Screening, with more than 50% of this score attributed to&#xD;
             non-ocular items.&#xD;
&#xD;
          7. Patients receiving steroids or pyridostigmine should not have any modification of drug&#xD;
             regimen during the month before Screening.&#xD;
&#xD;
          8. Female patients of childbearing potential must have a negative pregnancy test (serum&#xD;
             human chorionic gonadotropin [HCG] at screening); and must practice an effective,&#xD;
             reliable contraceptive regimen during the study and for up to 30 days following&#xD;
             discontinuation of treatment.&#xD;
&#xD;
          9. Ability to participate in the study based on overall health of the patient and disease&#xD;
             prognosis, as applicable, in the opinion of the Investigator; and able to comply with&#xD;
             all requirements of the protocol, including completion of study questionnaires.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Epilepsy and currently on medication.&#xD;
&#xD;
          2. Concomitant use of medicinal products with a known potential to cause QTc&#xD;
             prolongation.&#xD;
&#xD;
          3. Patients with long QT syndromes.&#xD;
&#xD;
          4. History of thymectomy within 12 months before Screening.&#xD;
&#xD;
          5. An electrocardiogram (ECG) within 6 months before starting treatment that shows&#xD;
             clinically significant abnormalities, in the opinion of the Investigator.&#xD;
&#xD;
          6. Breastfeeding or pregnant at Screening or planning to become pregnant at any time&#xD;
             during the study.&#xD;
&#xD;
          7. Patients receiving immunomodulatory treatment (e.g. plasma exchange [PE], therapeutic&#xD;
             plasma exchange [TPE], intravenous immunoglobulin G [IVIG]) should not have any&#xD;
             treatment in the previous 4 weeks prior to Randomization or at any time during the&#xD;
             study.&#xD;
&#xD;
          8. Use of rituximab or other similar biologic medications for immunomodulation within 6&#xD;
             months prior to Screening.&#xD;
&#xD;
          9. Treatment with an investigational drug (other than amifampridine) or device within 30&#xD;
             days before Screening or while participating in this study.&#xD;
&#xD;
         10. Any medical condition that, in the opinion of the Investigator, might interfere with&#xD;
             the patient's participation in the study, poses an added risk for the patient, or&#xD;
             confound the assessment of the patient.&#xD;
&#xD;
         11. History of drug allergy to any pyridine-containing substances or any amifampridine&#xD;
             excipient(s).&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Renato Mantegazza, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Carlo Besta Neurologic Institute</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Cleveland Clinic</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44195</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Univerity of Pennsylvania</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>July 2021</verification_date>
  <study_first_submitted>September 28, 2017</study_first_submitted>
  <study_first_submitted_qc>October 2, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">October 6, 2017</study_first_posted>
  <results_first_submitted>April 13, 2021</results_first_submitted>
  <results_first_submitted_qc>July 21, 2021</results_first_submitted_qc>
  <results_first_posted type="Actual">August 13, 2021</results_first_posted>
  <last_update_submitted>July 21, 2021</last_update_submitted>
  <last_update_submitted_qc>July 21, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">August 13, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>MuSK antibody positive</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Myasthenia Gravis</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Amifampridine</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>June 27, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/54/NCT03304054/Prot_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Statistical Analysis Plan</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>January 27, 2020</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/54/NCT03304054/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <recruitment_details>The study was conducted from 18 April 2018 - 24 April 2020 at 26 sites in the United States and Europe.</recruitment_details>
      <pre_assignment_details>A screening visit was conducted to ensure that each patient met inclusion/exclusion criteria. Those patients successfully completing screening had procedures/assessments conducted at the start of the Run-in period (Day 1, before starting study medication) and during run-in, until stable dose and frequency of amifampridine is established for at least 7 days, and at least a 2-point improvement in MG-ADL score was achieved from the start of Run-in to be eligible for randomization (Day 0).</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>Amifampridine Phosphate - MuSK</title>
          <description>Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day&#xD;
MuSK - patients diagnosed with MuSK-MG</description>
        </group>
        <group group_id="P2">
          <title>Amifampridine Phosphate - AChR-MG</title>
          <description>Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day&#xD;
AChR-MG- patients diagnosed with AChR-MG</description>
        </group>
        <group group_id="P3">
          <title>Placebo - MuSk</title>
          <description>Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day&#xD;
MuSK - patients diagnosed with MuSK-MG</description>
        </group>
        <group group_id="P4">
          <title>Placebo - AChR-MG</title>
          <description>Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day&#xD;
AChR-MG- patients diagnosed with AChR-MG</description>
        </group>
        <group group_id="P5">
          <title>Amifampridine Phosphate Only - MuSK</title>
          <description>Patients receiving amifampridine during the open-label run-in period but were not randomized for the crossover portion of the study and diagnosed with MuSK-MG.</description>
        </group>
        <group group_id="P6">
          <title>Amifampridine Phosphate Only- AChR-MG</title>
          <description>Patients receiving amifampridine during the open-label run-in period but were not randomized for the crossover portion of the study and diagnosed with AChR-MG.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="27"/>
                <participants group_id="P2" count="7"/>
                <participants group_id="P3" count="28"/>
                <participants group_id="P4" count="8"/>
                <participants group_id="P5" count="17"/>
                <participants group_id="P6" count="6"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="27"/>
                <participants group_id="P2" count="7"/>
                <participants group_id="P3" count="27"/>
                <participants group_id="P4" count="8"/>
                <participants group_id="P5" count="0"/>
                <participants group_id="P6" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
                <participants group_id="P3" count="1"/>
                <participants group_id="P4" count="0"/>
                <participants group_id="P5" count="17"/>
                <participants group_id="P6" count="6"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>The analysis of baseline characteristics included all patients in the Safety population, who are those patients who enrolled and received at least one dose of amifampridine. Patients who began the run-in period belong to the Safety population whether they were randomized to treatment or not.</population>
      <group_list>
        <group group_id="B1">
          <title>Amifampridine Phosphate</title>
          <description>Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day</description>
        </group>
        <group group_id="B2">
          <title>Placebo</title>
          <description>Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day</description>
        </group>
        <group group_id="B3">
          <title>Amifampridine Phosphate Only</title>
          <description>Patients receiving Amifampridine during the open-label Run-in period but were not randomized for the crossover portion of the study.</description>
        </group>
        <group group_id="B4">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="34"/>
            <count group_id="B2" value="36"/>
            <count group_id="B3" value="16"/>
            <count group_id="B4" value="86"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="53.1" spread="14.92"/>
                    <measurement group_id="B2" value="53.5" spread="12.61"/>
                    <measurement group_id="B3" value="45.9" spread="11.31"/>
                    <measurement group_id="B4" value="51.9" spread="13.52"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="24"/>
                    <measurement group_id="B2" value="28"/>
                    <measurement group_id="B3" value="14"/>
                    <measurement group_id="B4" value="66"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="10"/>
                    <measurement group_id="B2" value="8"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="20"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="15"/>
                    <measurement group_id="B4" value="18"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="33"/>
                    <measurement group_id="B2" value="34"/>
                    <measurement group_id="B3" value="1"/>
                    <measurement group_id="B4" value="68"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="1"/>
                    <measurement group_id="B4" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="7"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="31"/>
                    <measurement group_id="B2" value="34"/>
                    <measurement group_id="B3" value="13"/>
                    <measurement group_id="B4" value="78"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Weight</title>
          <units>kg</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="84.6" spread="21.11"/>
                    <measurement group_id="B2" value="84.3" spread="27.27"/>
                    <measurement group_id="B3" value="76.1" spread="32.28"/>
                    <measurement group_id="B4" value="82.9" spread="25.96"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Height</title>
          <units>cm</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="164.7" spread="7.64"/>
                    <measurement group_id="B2" value="163.5" spread="10.75"/>
                    <measurement group_id="B3" value="163.6" spread="7.37"/>
                    <measurement group_id="B4" value="164.0" spread="8.99"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results</title>
        <description>Myasthenia Gravis-Activities of Daily Living (MG-ADL) is a self-report scale to assess the patient's MG symptoms and functional performance of activities of daily living. The eight items are scored on a scale of 0-3 with 3 representing the most severe symptoms or impaired performance and 0 representing no symptoms or impaired performance. Each item was assessed by the patient at the last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total MG-ADL score was calculated as the sum of each item score, with a maximum score of 24 (most severe symptoms/impairment) and minimum score of 0 (least severe symptoms/impairment). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnos</description>
        <time_frame>Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time point at which a patient discontinued treatment early).</time_frame>
        <population>The analysis of primary outcome data was based on the Full Analysis Set population, which included all randomized patients who received at least one dose of study medication (amifampridine or placebo post randomization) and had at least one post-treatment efficacy assessment. Patients who discontinued with no post-randomization data (no Day 0 and no Day 10 data) were excluded from all efficacy analyses but were included in the safety analyses.</population>
        <group_list>
          <group group_id="O1">
            <title>Amifampridine Phosphate - MuSK</title>
            <description>Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day&#xD;
Patients randomized to receive Amifampridine and diagnosed with MuSK-MG.</description>
          </group>
          <group group_id="O2">
            <title>Placebo - MuSK</title>
            <description>Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day&#xD;
Patients randomized to receive Placebo and diagnosed with MuSK-MG.</description>
          </group>
          <group group_id="O3">
            <title>Amifampridine Phosphate - AChR</title>
            <description>Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day&#xD;
Patients randomized to receive Amifampridine and diagnosed with AChR-MG.</description>
          </group>
          <group group_id="O4">
            <title>Placebo - AChR</title>
            <description>Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day&#xD;
Patients randomized to receive Placebo and diagnosed with AChR-MG.</description>
          </group>
        </group_list>
        <measure>
          <title>Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results</title>
          <description>Myasthenia Gravis-Activities of Daily Living (MG-ADL) is a self-report scale to assess the patient's MG symptoms and functional performance of activities of daily living. The eight items are scored on a scale of 0-3 with 3 representing the most severe symptoms or impaired performance and 0 representing no symptoms or impaired performance. Each item was assessed by the patient at the last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total MG-ADL score was calculated as the sum of each item score, with a maximum score of 24 (most severe symptoms/impairment) and minimum score of 0 (least severe symptoms/impairment). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnos</description>
          <population>The analysis of primary outcome data was based on the Full Analysis Set population, which included all randomized patients who received at least one dose of study medication (amifampridine or placebo post randomization) and had at least one post-treatment efficacy assessment. Patients who discontinued with no post-randomization data (no Day 0 and no Day 10 data) were excluded from all efficacy analyses but were included in the safety analyses.</population>
          <units>Score on a Scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="27"/>
                <count group_id="O2" value="28"/>
                <count group_id="O3" value="7"/>
                <count group_id="O4" value="8"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline (Day 0) MG-ADL Total Raw Score</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.96" spread="2.915"/>
                    <measurement group_id="O2" value="3.86" spread="2.103"/>
                    <measurement group_id="O3" value="6.14" spread="3.388"/>
                    <measurement group_id="O4" value="7.00" spread="4.175"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Post-Baseline(Day 10 or time at which a patient discontinued treatment) MG-ADL Total Raw Score</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="6.00" spread="3.658"/>
                    <measurement group_id="O2" value="6.11" spread="3.19"/>
                    <measurement group_id="O3" value="4.71" spread="3.147"/>
                    <measurement group_id="O4" value="10.38" spread="3.583"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>CFB MG-ADL Total Score</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.04" spread="2.980"/>
                    <measurement group_id="O2" value="2.25" spread="3.658"/>
                    <measurement group_id="O3" value="-1.43" spread="2.225"/>
                    <measurement group_id="O4" value="3.38" spread="2.825"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
              <group_id>O3</group_id>
              <group_id>O4</group_id>
            </group_id_list>
            <groups_desc>A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnosed with MuSK-MG treated with amifampridine and placebo was conducted.</groups_desc>
            <non_inferiority_type>Other</non_inferiority_type>
            <p_value>0.2196</p_value>
            <p_value_desc>P-value for Wilcoxon-Mann-Whitney Test of Equality of change from baseline distributions between the amifampridine phosphate and placebo treatment groups.</p_value_desc>
            <method>Wilcoxon (Mann-Whitney)</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Quantitative Myasthenia Gravis (QMG) Total Score Summary Statistics by Time Point and MG Type: Wilcoxon-Mann-Whitney Rank Sum Test Results</title>
        <description>Quantitative Myasthenia Gravis (QMG) assesses the patient's general body strength and fatigability. Each test item is scored on a scale of 0-3 with 3 representing the most severe symptom results and 0 representing no symptom results. Each item was assessed by the patient at Screening, the first (Day 1) and last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total QMG score was calculated as the sum of each item score, with a maximum score of 39 (most severe symptoms) and minimum score of 0 (least severe symptoms). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnosed with MuSK-MG treated with amifampridine and placebo was conducted.</description>
        <time_frame>Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time at which a patient discontinued treatment early).</time_frame>
        <population>The analysis of secondary outcome data was based on the Full Analysis Set population, which included all randomized patients who received at least one dose of study medication (amifampridine or placebo post randomization) and had at least one post-treatment efficacy assessment. Patients who discontinued with no post-randomization data (no Day 0 and no Day 10 data) were excluded from all efficacy analyses but were included in the safety analyses.</population>
        <group_list>
          <group group_id="O1">
            <title>Amifampridine Phosphate - MuSK</title>
            <description>Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day&#xD;
Patients randomized to receive Amifampridine and diagnosed with MuSK-MG.</description>
          </group>
          <group group_id="O2">
            <title>Placebo - MuSK</title>
            <description>Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day&#xD;
Patients randomized to receive Placebo and diagnosed with MuSK-MG.</description>
          </group>
          <group group_id="O3">
            <title>Amifampridine Phosphate - AChR</title>
            <description>Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day&#xD;
Patients randomized to receive Amifampridine and diagnosed with AChR-MG.</description>
          </group>
          <group group_id="O4">
            <title>Placebo - AChR</title>
            <description>Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day&#xD;
Patients randomized to receive Placebo and diagnosed with AChR-MG.</description>
          </group>
        </group_list>
        <measure>
          <title>Quantitative Myasthenia Gravis (QMG) Total Score Summary Statistics by Time Point and MG Type: Wilcoxon-Mann-Whitney Rank Sum Test Results</title>
          <description>Quantitative Myasthenia Gravis (QMG) assesses the patient's general body strength and fatigability. Each test item is scored on a scale of 0-3 with 3 representing the most severe symptom results and 0 representing no symptom results. Each item was assessed by the patient at Screening, the first (Day 1) and last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total QMG score was calculated as the sum of each item score, with a maximum score of 39 (most severe symptoms) and minimum score of 0 (least severe symptoms). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnosed with MuSK-MG treated with amifampridine and placebo was conducted.</description>
          <population>The analysis of secondary outcome data was based on the Full Analysis Set population, which included all randomized patients who received at least one dose of study medication (amifampridine or placebo post randomization) and had at least one post-treatment efficacy assessment. Patients who discontinued with no post-randomization data (no Day 0 and no Day 10 data) were excluded from all efficacy analyses but were included in the safety analyses.</population>
          <units>Score on a Scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="27"/>
                <count group_id="O2" value="28"/>
                <count group_id="O3" value="7"/>
                <count group_id="O4" value="8"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline (Day 0) QMG Total Raw Score</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="10.00" spread="3.873"/>
                    <measurement group_id="O2" value="8.64" spread="3.744"/>
                    <measurement group_id="O3" value="10.57" spread="3.359"/>
                    <measurement group_id="O4" value="14.13" spread="4.155"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Post-Baseline (day 10 or time at which a patient discontinued treatment early) QMG Total Raw Score</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="11.19" spread="5.367"/>
                    <measurement group_id="O2" value="10.16" spread="3.648"/>
                    <measurement group_id="O3" value="10.86" spread="3.024"/>
                    <measurement group_id="O4" value="14.43" spread="3.952"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>CFB QMG Total Score</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.19" spread="3.99"/>
                    <measurement group_id="O2" value="1.80" spread="3.948"/>
                    <measurement group_id="O3" value="0.29" spread="3.039"/>
                    <measurement group_id="O4" value="0.86" spread="2.673"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
              <group_id>O3</group_id>
              <group_id>O4</group_id>
            </group_id_list>
            <groups_desc>A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnosed with MuSK-MG treated with amifampridine and placebo was conducted.</groups_desc>
            <non_inferiority_type>Other</non_inferiority_type>
            <p_value>0.3736</p_value>
            <p_value_desc>P-value for Wilcoxon-Mann-Whitney Test of Equality of change from baseline distributions between the amifampridine phosphate and placebo treatment groups.</p_value_desc>
            <method>Wilcoxon (Mann-Whitney)</method>
          </analysis>
        </analysis_list>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Serious adverse events (SAEs) were recorded for each patient from the time informed consent was obtained at Screening and continues through four weeks after the last dose or at the early discontinuation visit, for a total of approximately 52 days. Non-serious AEs were recorded for each patient after the first administration of study drug through the last visit or at the early discontinuation visit, for a total of approximately 10 days.</time_frame>
      <desc>Serious classification based on the FDA regulatory definition of a serious AE.</desc>
      <group_list>
        <group group_id="E1">
          <title>Amifampridine Phosphate</title>
          <description>Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day.&#xD;
The AEs reported within this treatment arm are those experienced by all participants who received Amifampridine Phosphate at the time of onset of the AE. Because this study has a run-in period where all subjects received Amifampridine Phosphate, the number of at-risk subjects in this treatment arm is identical to the overall number of at-risk participants. That is, the at-risk count includes those randomized to the Amifampridine Phosphate-Placebo sequence, Placebo - Amifampridine Phosphate sequence and those that received Amifampridine Phosphate during the open-label Run-in period but were not randomized for the crossover portion of the study.</description>
        </group>
        <group group_id="E2">
          <title>Placebo</title>
          <description>Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day&#xD;
The AEs reported within this treatment arm are those experienced by participants who received Placebo at the time of onset of the AE. This includes those randomized to the Placebo - Amifampridine Phosphate sequence.</description>
        </group>
        <group group_id="E3">
          <title>Overall</title>
          <description>Includes all participants who received study drug, including Amifampridine Phosphate during the open-label Run-in period or double-blind treatment period or Placebo during the double-blind treatment period.</description>
        </group>
      </group_list>
      <serious_events>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Myasthenia gravis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Myasthenia gravis crisis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>2</frequency_threshold>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="80" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="7" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="80" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Palpitations</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Ear and labyrinth disorders</title>
            <event_list>
              <event>
                <sub_title>Tinnitus</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Eye disorders</title>
            <event_list>
              <event>
                <sub_title>Diplopia</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal discomfort</sub_title>
                <counts group_id="E1" subjects_affected="5" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="5" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Abdominal pain upper</sub_title>
                <counts group_id="E1" subjects_affected="7" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="7" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" subjects_affected="12" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="12" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Dyspepsia</sub_title>
                <counts group_id="E1" subjects_affected="7" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="7" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Hypoaesthesia oral</sub_title>
                <counts group_id="E1" subjects_affected="9" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="9" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" subjects_affected="14" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="14" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Paraesthesia oral</sub_title>
                <counts group_id="E1" subjects_affected="37" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="37" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Abdominal pain</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Vomiting</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" subjects_affected="10" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="12" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Asthenia</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Feeling cold</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Feeling hot</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Ear infection</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Hordeolum</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Sinusitis</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Urinary tract infection</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Fall</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Muscle spasms</sub_title>
                <counts group_id="E1" subjects_affected="5" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="5" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Back pain</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Mastication disorder</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Muscular weakness</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Pain in extremity</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="5" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Dizziness</sub_title>
                <counts group_id="E1" subjects_affected="8" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="8" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" subjects_affected="11" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="11" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Paraesthesia</sub_title>
                <counts group_id="E1" subjects_affected="34" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="34" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Hypoaesthesia</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="4" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Myasthenia gravis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Sensory disturbance</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Speech disorder</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Anxiety</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Insomnia</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Dyspnoea</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="4" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Oropharyngeal pain</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="4" subjects_at_risk="86"/>
              </event>
              <event>
                <sub_title>Rhinorrhoea</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Rash maculo-papular</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Vascular disorders</title>
            <event_list>
              <event>
                <sub_title>Peripheral coldness</sub_title>
                <counts group_id="E1" subjects_affected="5" subjects_at_risk="86"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="36"/>
                <counts group_id="E3" subjects_affected="5" subjects_at_risk="86"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Gary Ingenito</name_or_title>
      <organization>Catalyst Pharmaceuticals, Inc.</organization>
      <phone>305-420-3200</phone>
      <email>gingenito@catalystpharma.com</email>
    </point_of_contact>
  </clinical_results>
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03304054
org study id: MSK-002
nct id: NCT03304054
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
Efficacy and safety of amifampridine phosphate in improving the activities of daily living
      for patients with antibody positive MuSK myasthenia gravis.
Randomized, double-blind, placebo-controlled, parallel group study is designed to evaluate
      the safety, tolerability and efficacy of amifampridine phosphate in patients with MuSK-MG. In
      addition, a sample of AChR-MG patients will be assess for efficacy and safety of
      amifampridine phosphate. Planned duration of participation for each patient is at least 38
      days, excluding the screening period. Eligible patients will be titrated to an efficacious
      dose of amifampridine phosphate and those who demonstrate improvement will be randomized to
      either placebo or amifampridine, in a double-blind fashion, for 10 days.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results
time frame: Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time point at which a patient discontinued treatment early).
description: Myasthenia Gravis-Activities of Daily Living (MG-ADL) is a self-report scale to assess the patient's MG symptoms and functional performance of activities of daily living. The eight items are scored on a scale of 0-3 with 3 representing the most severe symptoms or impaired performance and 0 representing no symptoms or impaired performance. Each item was assessed by the patient at the last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total MG-ADL score was calculated as the sum of each item score, with a maximum score of 24 (most severe symptoms/impairment) and minimum score of 0 (least severe symptoms/impairment). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnos
measure: Quantitative Myasthenia Gravis (QMG) Total Score Summary Statistics by Time Point and MG Type: Wilcoxon-Mann-Whitney Rank Sum Test Results
time frame: Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time at which a patient discontinued treatment early).
description: Quantitative Myasthenia Gravis (QMG) assesses the patient's general body strength and fatigability. Each test item is scored on a scale of 0-3 with 3 representing the most severe symptom results and 0 representing no symptom results. Each item was assessed by the patient at Screening, the first (Day 1) and last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total QMG score was calculated as the sum of each item score, with a maximum score of 39 (most severe symptoms) and minimum score of 0 (least severe symptoms). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnosed with MuSK-MG treated with amifampridine and placebo was conducted.
arm group label: amifamapridine phosphate tablets
arm group type: Experimental
arm group label: placebo tablets
arm group type: Placebo Comparator
intervention type: Drug
intervention name: Amifampridine Phosphate
description: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day
arm group label: amifamapridine phosphate tablets
intervention type: Drug
intervention name: Placebo Oral Tablet
description: tablets matching amifampridine phosphate, 3 to 4 times a day
arm group label: placebo tablets
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Renato Mantegazza, MD
role: Principal Investigator
affiliation: Carlo Besta Neurologic Institute
facility: 
facility: 
country: United States
responsible party type: Sponsor
mesh term: Myasthenia Gravis
mesh term: Amifampridine
sharing ipd: No
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 