<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/NCT02191826</url>
  </required_header>
  <id_info>
    <org_study_id>SOMCT01-C</org_study_id>
    <secondary_id>2014-001586-27</secondary_id>
    <nct_id>NCT02191826</nct_id>
  </id_info>
  <brief_title>Study of SOM0226 in Familial Amyloid Polyneuropathy</brief_title>
  <official_title>Study of SOM0226 in Familial Amyloid Polyneuropathy (FAP) Patients and Asymptomatic Carriers to Evaluate Protein Stabilization Activity</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>SOM Innovation Biotech SA</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Hospital Vall d'Hebron</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>SOM Innovation Biotech SA</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is an open label interventional Phase IIa proof of concept clinical trial designed in&#xD;
      two phases separated by a washout period of 6 weeks (± 2 weeks) , to evaluate the TTR&#xD;
      stabilization activity of SOM0226 in healthy volunteers (wild type), TTR-FAP patients and&#xD;
      asymptomatic carriers.&#xD;
&#xD;
        -  Phase A (24 hours): SOM0226 single dose&#xD;
&#xD;
        -  Phase B (32 hours): SOM0226 multiple dose&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>July 2014</start_date>
  <completion_date type="Actual">November 2015</completion_date>
  <primary_completion_date type="Actual">November 2015</primary_completion_date>
  <phase>Phase 1/Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>TTR stabilization</measure>
    <time_frame>24 hours and 32 hours</time_frame>
    <description>TTR stabilization will be measured by determining the concentration of tetrameric TTR after denaturation. The ratio of TTR tetramer after denaturation / TTR tetramer before denaturation of plasma samples from treated subjects will be compared to their baseline ratio. TTR stabilization will be determined as percentage of the baseline ratio.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Pharmacodynamics assessment</measure>
    <time_frame>24 hours and 32 hours</time_frame>
    <description>Determine the minimal molar ratio SOM0226:TTR that must be reached in plasma to confer maximal TTR stabilization</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Safety</measure>
    <time_frame>24 hours</time_frame>
    <description>Drug safety will be assessed by blood biochemistry and hematology analysis, patient exploration and adverse event reporting</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">17</enrollment>
  <condition>Familial Amyloid Polyneuropathy (FAP)</condition>
  <arm_group>
    <arm_group_label>SOM0226 single dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>SOM0226 multiple doses</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>SOM0226</intervention_name>
    <description>Oral</description>
    <arm_group_label>SOM0226 multiple doses</arm_group_label>
    <arm_group_label>SOM0226 single dose</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Participant is willing and able to give informed consent for participation in the&#xD;
             study&#xD;
&#xD;
          -  Male or Female, aged 18 years or above at the time of consent&#xD;
&#xD;
          -  Two cohorts defined by TTR variant (wild type: healthy volunteers; TTR mutant:&#xD;
             asymptomatic carriers with documented mutation in TTR or diagnosed TTR-FAP patients&#xD;
             (stage 1 or 2) not undergoing pharmacological treatment with Vyndaqel&#xD;
&#xD;
          -  Body Mass Index (BMI) &gt; 17.5 kg/m2&#xD;
&#xD;
          -  Female participants of child bearing potential and male participants whose partner is&#xD;
             of child bearing potential must be willing to ensure that they or their partner use&#xD;
             effective contraception during the study and for 2 month thereafter&#xD;
&#xD;
          -  Able (in the Investigators opinion) and willing to comply with all study requirements&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Female participant who is pregnant, lactating or planning pregnancy during the course&#xD;
             of the study&#xD;
&#xD;
          -  Evidence of history of clinically significant hepatic disease&#xD;
&#xD;
          -  An ALT or AST measurement &gt; 2 times the ULN (Upper Limit of Normal)&#xD;
&#xD;
          -  Scheduled elective surgery or other procedures requiring general anaesthesia during&#xD;
             the study&#xD;
&#xD;
          -  Donation of blood during the study or within the past 4 weeks&#xD;
&#xD;
          -  Treatment (during the study or within the past 4 weeks) with a prescription or&#xD;
             investigational drug for the treatment of TTR amyloidosis&#xD;
&#xD;
          -  Treatment with NSAIDs (nonsteroidal antiinflammatory drug) during the study or within&#xD;
             the past 4 weeks. The following NSAID are allowed: acetylsalicylic acid, etodolac,&#xD;
             ibuprofen, indomethacin, ketoprofen, nabumetone, naproxen, nimesulide, piroxicam and&#xD;
             sulindac&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Josep Gámez, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Hospital Vall d'Hebron</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Hospital Vall d'Hebron</name>
      <address>
        <city>Barcelona</city>
        <state>Catalonia</state>
        <zip>08035</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Spain</country>
  </location_countries>
  <link>
    <url>http://www.sombiotech.com</url>
    <description>Related Info</description>
  </link>
  <verification_date>November 2016</verification_date>
  <study_first_submitted>July 9, 2014</study_first_submitted>
  <study_first_submitted_qc>July 14, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">July 16, 2014</study_first_posted>
  <last_update_submitted>November 29, 2016</last_update_submitted>
  <last_update_submitted_qc>November 29, 2016</last_update_submitted_qc>
  <last_update_posted type="Estimate">November 30, 2016</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Transthyretin</keyword>
  <keyword>TTR</keyword>
  <keyword>Amyloidosis</keyword>
  <keyword>SOM0226</keyword>
  <keyword>Familiar Amyloid Polyneuropathy</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Polyneuropathies</mesh_term>
    <mesh_term>Amyloid Neuropathies</mesh_term>
    <mesh_term>Amyloid Neuropathies, Familial</mesh_term>
    <mesh_term>Amyloidosis</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Tolcapone</mesh_term>
  </intervention_browse>
  <!-- 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/NCT02191826
org study id: SOMCT01-C
secondary id: 2014-001586-27
nct id: NCT02191826
lead sponsor: 
collaborator: 
has dmc: No
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
This is an open label interventional Phase IIa proof of concept clinical trial designed in
      two phases separated by a washout period of 6 weeks (± 2 weeks) , to evaluate the TTR
      stabilization activity of SOM0226 in healthy volunteers (wild type), TTR-FAP patients and
      asymptomatic carriers.

        -  Phase A (24 hours): SOM0226 single dose

        -  Phase B (32 hours): SOM0226 multiple dose
allocation: Non-Randomized
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: TTR stabilization
time frame: 24 hours and 32 hours
description: TTR stabilization will be measured by determining the concentration of tetrameric TTR after denaturation. The ratio of TTR tetramer after denaturation / TTR tetramer before denaturation of plasma samples from treated subjects will be compared to their baseline ratio. TTR stabilization will be determined as percentage of the baseline ratio.
measure: Pharmacodynamics assessment
time frame: 24 hours and 32 hours
description: Determine the minimal molar ratio SOM0226:TTR that must be reached in plasma to confer maximal TTR stabilization
measure: Safety
time frame: 24 hours
description: Drug safety will be assessed by blood biochemistry and hematology analysis, patient exploration and adverse event reporting
arm group label: SOM0226 single dose
arm group type: Experimental
arm group label: SOM0226 multiple doses
arm group type: Experimental
intervention type: Drug
intervention name: SOM0226
description: Oral
arm group label: SOM0226 multiple doses
arm group label: SOM0226 single dose
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: Accepts Healthy Volunteers
last name: Josep Gámez, MD
role: Principal Investigator
affiliation: Hospital Vall d'Hebron
facility: 
country: Spain
url: http://www.sombiotech.com
description: Related Info
responsible party type: Sponsor
mesh term: Polyneuropathies
mesh term: Amyloid Neuropathies
mesh term: Amyloid Neuropathies, Familial
mesh term: Amyloidosis
mesh term: Tolcapone