<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/NCT01707264</url>
  </required_header>
  <id_info>
    <org_study_id>NEOD001-001</org_study_id>
    <nct_id>NCT01707264</nct_id>
  </id_info>
  <brief_title>Phase 1/2, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis</brief_title>
  <official_title>A Phase 1/2, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Prothena Biosciences Ltd.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Prothena Biosciences Ltd.</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Dose escalation study to determine the maximum tolerated dose of NEOD001 in approximately 30&#xD;
      subjects with AL amyloidosis. Expansion phase to evaluate safety, efficacy and&#xD;
      pharmacokinetics of NEOD001 in 25 additional subjects at the maximum tolerated dose.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The purpose of the dose escalation phase of the study is to determine the maximum tolerated&#xD;
      dose/Phase 2 recommended dose of NEOD001 when given as a single agent intravenously in&#xD;
      approximately 30 subjects with AL amyloidosis.&#xD;
&#xD;
      The purpose of the expansion phase of the study is to evaluate the safety, preliminary&#xD;
      efficacy and pharmacokinetics of single agent NEOD001 at the maximum tolerated dose/Phase 2&#xD;
      recommended dose in approximately 25 additional evaluable subjects.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">April 2013</start_date>
  <completion_date type="Actual">August 9, 2016</completion_date>
  <primary_completion_date type="Actual">August 9, 2016</primary_completion_date>
  <phase>Phase 1/Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <intervention_model>Sequential Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Safety and tolerability</measure>
    <time_frame>28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator</time_frame>
    <description>Adverse event profile&#xD;
Dose limiting toxicity and maximum tolerated dose</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Maximum tolerated dose</measure>
    <time_frame>28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator</time_frame>
    <description>Adverse event profile&#xD;
Dose Limiting Toxicity and maximum tolerated dose</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Pharmacokinetics</measure>
    <time_frame>28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator</time_frame>
    <description>• Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Immunogenicity</measure>
    <time_frame>28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator</time_frame>
    <description>• Measurement of anti-NEOD001 antibodies</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Hematologic Response</measure>
    <time_frame>28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator</time_frame>
    <description>• Hematologic response</description>
  </other_outcome>
  <other_outcome>
    <measure>Organ response</measure>
    <time_frame>28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator</time_frame>
    <description>Organ response&#xD;
Changes in organ function markers</description>
  </other_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">69</enrollment>
  <condition>Primary Amyloidosis</condition>
  <arm_group>
    <arm_group_label>NEOD001</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>NEOD001 will be administered intravenously once every 28 days. The starting dose will be 0.5 mg/kg. Dose escalation will continue until the the maximum tolerated dose is determined for single agent NEOD001. Approximately 20 additional subjects will be treated with the maximum tolerated dose.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>NEOD001</intervention_name>
    <description>Monoclonal antibody administered by intravenous infusion every 28 days.</description>
    <arm_group_label>NEOD001</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Males and females aged ≥18 years;&#xD;
&#xD;
          2. ECOG performance status (PS) 0-2;&#xD;
&#xD;
          3. Diagnosis of systemic AL amyloidosis (subjects with non-AL amyloidosis are not&#xD;
             eligible);&#xD;
&#xD;
          4. Received at least one prior systemic therapy, which may include stem cell transplant,&#xD;
             for AL amyloidosis;&#xD;
&#xD;
          5. Have adequate organ function;&#xD;
&#xD;
          6. Ability to understand and willingness to sign informed consent prior to initiation of&#xD;
             any study procedures.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Secondary or familial amyloidosis;&#xD;
&#xD;
          2. Life expectancy of &lt; 3 months;&#xD;
&#xD;
          3. Symptomatic multiple myeloma;&#xD;
&#xD;
          4. Hypersensitivities to other monoclonal antibodies;&#xD;
&#xD;
          5. Known HIV infection;&#xD;
&#xD;
          6. Women who are lactating;&#xD;
&#xD;
          7. Any other condition or prior therapy, which in the opinion of the PI, would make the&#xD;
             subject unsuitable for the study.&#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>Julia Martinisi</last_name>
    <role>Study Director</role>
    <affiliation>Prothena Biosciences Ltd.</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Stanford University Cancer Center</name>
      <address>
        <city>Palo Alto</city>
        <state>California</state>
        <zip>94305</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tufts Medical Center</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02111</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Boston University School of Medicine</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02118</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Karmanos Cancer Institute</name>
      <address>
        <city>Detroit</city>
        <state>Michigan</state>
        <zip>48201</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mayo Clinic</name>
      <address>
        <city>Rochester</city>
        <state>Minnesota</state>
        <zip>55905</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Memorial Sloan-Kettering Cancer Center</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10065</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital of the University 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>August 2018</verification_date>
  <study_first_submitted>October 11, 2012</study_first_submitted>
  <study_first_submitted_qc>October 12, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 16, 2012</study_first_posted>
  <disposition_first_submitted>November 20, 2017</disposition_first_submitted>
  <disposition_first_submitted_qc>November 20, 2017</disposition_first_submitted_qc>
  <disposition_first_posted type="Actual">November 22, 2017</disposition_first_posted>
  <last_update_submitted>August 24, 2018</last_update_submitted>
  <last_update_submitted_qc>August 24, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">August 28, 2018</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>AL amyloidosis</keyword>
  <keyword>Primary amyloidosis</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Immunoglobulin Light-chain Amyloidosis</mesh_term>
    <mesh_term>Amyloidosis</mesh_term>
  </condition_browse>
  <!-- Results have not yet been posted, but a results disposition has been submitted           -->
</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/NCT01707264
org study id: NEOD001-001
nct id: NCT01707264
lead sponsor: 
has dmc: Yes
Dose escalation study to determine the maximum tolerated dose of NEOD001 in approximately 30
      subjects with AL amyloidosis. Expansion phase to evaluate safety, efficacy and
      pharmacokinetics of NEOD001 in 25 additional subjects at the maximum tolerated dose.
The purpose of the dose escalation phase of the study is to determine the maximum tolerated
      dose/Phase 2 recommended dose of NEOD001 when given as a single agent intravenously in
      approximately 30 subjects with AL amyloidosis.

      The purpose of the expansion phase of the study is to evaluate the safety, preliminary
      efficacy and pharmacokinetics of single agent NEOD001 at the maximum tolerated dose/Phase 2
      recommended dose in approximately 25 additional evaluable subjects.
intervention model: Sequential Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Safety and tolerability
time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
description: Adverse event profile
Dose limiting toxicity and maximum tolerated dose
measure: Maximum tolerated dose
time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
description: Adverse event profile
Dose Limiting Toxicity and maximum tolerated dose
measure: Pharmacokinetics
time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
description: • Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz
measure: Immunogenicity
time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
description: • Measurement of anti-NEOD001 antibodies
measure: Hematologic Response
time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
description: • Hematologic response
measure: Organ response
time frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator
description: Organ response
Changes in organ function markers
arm group label: NEOD001
arm group type: Experimental
description: NEOD001 will be administered intravenously once every 28 days. The starting dose will be 0.5 mg/kg. Dose escalation will continue until the the maximum tolerated dose is determined for single agent NEOD001. Approximately 20 additional subjects will be treated with the maximum tolerated dose.
intervention type: Drug
intervention name: NEOD001
description: Monoclonal antibody administered by intravenous infusion every 28 days.
arm group label: NEOD001
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Julia Martinisi
role: Study Director
affiliation: Prothena Biosciences Ltd.
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: United States
responsible party type: Sponsor
mesh term: Immunoglobulin Light-chain Amyloidosis
mesh term: Amyloidosis