<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/NCT03819660</url>
  </required_header>
  <id_info>
    <org_study_id>SMA-002</org_study_id>
    <nct_id>NCT03819660</nct_id>
  </id_info>
  <brief_title>Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3</brief_title>
  <acronym>SMA3</acronym>
  <official_title>Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3</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>
      A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy&#xD;
      (SMA) Type 3.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This open-label, long term safety study is designed to evaluate the safety of amifampridine&#xD;
      phosphate in ambulatory patients diagnosed SMA Type 3 over an extended period. The study is&#xD;
      planned to include approximately 12 male and female SMA Type 3 patients. The planned duration&#xD;
      of participation for each patient may be up to 1 year. Patients must have demonstrated&#xD;
      benefit during the initial SMA-001 trial.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Development of indication not being pursued&#xD;
  </why_stopped>
  <start_date type="Actual">March 7, 2019</start_date>
  <completion_date type="Actual">September 13, 2021</completion_date>
  <primary_completion_date type="Actual">September 13, 2021</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <intervention_model_description>Open-label, long term safety treatment</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>The number and severity of treatment related adverse events and serious adverse events as assessed by CTCAE v4.0</measure>
    <time_frame>18 months</time_frame>
    <description>Safety analyses will be conducted on the safety population (i.e. all patients who receive at least 1 dose of amifampridine. The safety analysis will be descriptive and will be presented on observed data only.&#xD;
All AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). The incidence of TEAEs will be summarized by system organ class, preferred term, relationship to treatment, and severity.</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">12</enrollment>
  <condition>Muscle Atrophy</condition>
  <arm_group>
    <arm_group_label>amifampridine phosphate</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Oral tablets, 15 to 80 mg per day in divided doses 3 to 4 times a day for up to 18 months.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Amifampridine Phosphate 10 MG Oral Tablet</intervention_name>
    <description>Oral tablets</description>
    <arm_group_label>amifampridine phosphate</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        Individuals eligible to participate in this study must meet all the following inclusion&#xD;
        criteria:&#xD;
&#xD;
          1. Participated in the SMA-001 study&#xD;
&#xD;
          2. 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;
          3. Female patients of childbearing potential must have a negative pregnancy test (urine&#xD;
             human chorionic gonadotropin [HCG] at the end of SMA-001 study); and must practice an&#xD;
             effective, reliable contraceptive regimen during the study and for up to 30 days&#xD;
             following discontinuation of treatment.&#xD;
&#xD;
          4. 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;
        CRITERIA FOR INCLUSION AND EXCLUSION:&#xD;
&#xD;
        Individuals eligible to participate in this study must meet all the following inclusion&#xD;
        criteria:&#xD;
&#xD;
          1. Participated in the SMA-001 study&#xD;
&#xD;
          2. 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;
          3. Female patients of childbearing potential must have a negative pregnancy test (urine&#xD;
             human chorionic gonadotropin [HCG] at the end of SMA-001 study); and must practice an&#xD;
             effective, reliable contraceptive regimen during the study and for up to 30 days&#xD;
             following discontinuation of treatment.&#xD;
&#xD;
          4. 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;
        Individuals who met any of the exclusion criteria in the original protocol or those listed&#xD;
        below are not eligible to participate in the study:&#xD;
&#xD;
          1. Epilepsy and currently on medication.&#xD;
&#xD;
          2. Uncontrolled asthma.&#xD;
&#xD;
          3. Concomitant use with sultopride.&#xD;
&#xD;
          4. Concomitant use with medicinal products with a narrow therapeutic window.&#xD;
&#xD;
          5. Concomitant use with medicinal products with a known to cause QTc prolongation.&#xD;
&#xD;
          6. Clinically significant abnormalities in 12 lead ECG, in the opinion of the&#xD;
             Investigator.&#xD;
&#xD;
          7. Subjects with congenital QT syndromes.&#xD;
&#xD;
          8. Breastfeeding or pregnant at Screening or planning to become pregnant at any time&#xD;
             during the study.&#xD;
&#xD;
          9. Intolerable amifampridine-related side effects&#xD;
&#xD;
         10. Treatment with an investigational drug (other than amifampridine) or device while&#xD;
             participating in this study.&#xD;
&#xD;
         11. 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;
         12. History of drug allergy to any pyridine-containing substances or any amifampridine&#xD;
             excipient(s).&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Years</minimum_age>
    <maximum_age>50 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Lorenzo Maggi, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Carlo Besta Institute, Milan, Italy</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Neurological Institute Carlo Besta</name>
      <address>
        <city>Milano</city>
        <state>Lombardy</state>
        <zip>20133</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Italy</country>
  </location_countries>
  <verification_date>March 2022</verification_date>
  <study_first_submitted>January 23, 2019</study_first_submitted>
  <study_first_submitted_qc>January 24, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 28, 2019</study_first_posted>
  <last_update_submitted>March 14, 2022</last_update_submitted>
  <last_update_submitted_qc>March 14, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">March 29, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Type 3</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Muscular Atrophy</mesh_term>
    <mesh_term>Muscular Atrophy, Spinal</mesh_term>
    <mesh_term>Atrophy</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Amifampridine</mesh_term>
  </intervention_browse>
  <pending_results>
    <submitted>September 1, 2023</submitted>
  </pending_results>
  <!-- Results have not yet been posted for this study                                          -->
</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/NCT03819660
org study id: SMA-002
nct id: NCT03819660
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy
      (SMA) Type 3.
This open-label, long term safety study is designed to evaluate the safety of amifampridine
      phosphate in ambulatory patients diagnosed SMA Type 3 over an extended period. The study is
      planned to include approximately 12 male and female SMA Type 3 patients. The planned duration
      of participation for each patient may be up to 1 year. Patients must have demonstrated
      benefit during the initial SMA-001 trial.
allocation: N/A
intervention model: Single Group Assignment
intervention model description: Open-label, long term safety treatment
primary purpose: Treatment
masking: None (Open Label)
measure: The number and severity of treatment related adverse events and serious adverse events as assessed by CTCAE v4.0
time frame: 18 months
description: Safety analyses will be conducted on the safety population (i.e. all patients who receive at least 1 dose of amifampridine. The safety analysis will be descriptive and will be presented on observed data only.
All AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). The incidence of TEAEs will be summarized by system organ class, preferred term, relationship to treatment, and severity.
arm group label: amifampridine phosphate
arm group type: Experimental
description: Oral tablets, 15 to 80 mg per day in divided doses 3 to 4 times a day for up to 18 months.
intervention type: Drug
intervention name: Amifampridine Phosphate 10 MG Oral Tablet
description: Oral tablets
arm group label: amifampridine phosphate
criteria: 
gender: All
minimum age: 6 Years
maximum age: 50 Years
healthy volunteers: No
last name: Lorenzo Maggi, MD
role: Principal Investigator
affiliation: Carlo Besta Institute, Milan, Italy
facility: 
country: Italy
responsible party type: Sponsor
mesh term: Muscular Atrophy
mesh term: Muscular Atrophy, Spinal
mesh term: Atrophy
mesh term: Amifampridine
submitted: September 1, 2023