<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/NCT03819647</url>
  </required_header>
  <id_info>
    <org_study_id>HYPOP (STP194)</org_study_id>
    <nct_id>NCT03819647</nct_id>
  </id_info>
  <brief_title>Evaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary Hyperoxaluria</brief_title>
  <official_title>Evaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary Hyperoxaluria</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Biocodex</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Biocodex</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
    <is_us_export>No</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      Evaluation of the efficacy of stiripentol (Diacomit) as monotherapy for the treatment of&#xD;
      primary hyperoxaluria.&#xD;
&#xD;
      Pilot clinical study, open, prospective and multicenter.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">May 21, 2019</start_date>
  <completion_date type="Actual">March 8, 2021</completion_date>
  <primary_completion_date type="Actual">December 18, 2020</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>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Relative variation (%) of the molar ratio [oxaluria / creatinuria] between baseline and after two weeks of treatment.</measure>
    <time_frame>Change (%) of the molar ratio [oxaluria / creatinuria] between the baseline value (average of 3 measures done during pre-treatment period) and the value (average of 2 measures) after 2 weeks of treatment.</time_frame>
    <description>Relative variation (%) of the molar ratio [oxaluria / creatinuria] between baseline and after two weeks of treatment.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Relative variation (%) of the molar ratio [oxaluria / creatinuria] between baseline and after three weeks of treatment.</measure>
    <time_frame>Change (%) of the molar ratio [oxaluria / creatinuria] between the baseline value (average of 3 measures done during pre-treatment period) and the value (average of 2 measures) after 3 weeks of treatment.</time_frame>
    <description>Relative variation (%) of the molar ratio [oxaluria / creatinuria] between baseline and after three weeks of treatment.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Response to treatment defined by a decrease&gt; 20% of the molar ratio [oxaluria / creatinuria]</measure>
    <time_frame>3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)</time_frame>
    <description>Response to treatment defined by a decrease&gt; 20% of the molar ratio [oxaluria / creatinuria]</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Relative variation (%) of supersaturation of urine with calcium oxalate between the start and the end of treatment period</measure>
    <time_frame>3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)</time_frame>
    <description>Relative variation (%) of supersaturation of urine with calcium oxalate between the start and the end of treatment period</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Relative variation (%) in overall crystalline volume measured by crystalluria on fresh urine between the start and the end of treatment period</measure>
    <time_frame>3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)</time_frame>
    <description>Relative variation (%) in overall crystalline volume measured by crystalluria on fresh urine between the start and the end of treatment period</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Effect of stiripentol dose increase on absolute decrease of the molar ratio [oxaluria / creatinuria]</measure>
    <time_frame>3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)</time_frame>
    <description>Effect of stiripentol dose increase on absolute decrease of the molar ratio [oxaluria / creatinuria]</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Effect of stiripentol dose increase on relative decrease (%) of the molar ratio [oxaluria / creatinuria]</measure>
    <time_frame>3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)</time_frame>
    <description>Effect of stiripentol dose increase on relative decrease (%) of the molar ratio [oxaluria / creatinuria]</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Blood test results (hepatic assessment) at the start and at the end of the study</measure>
    <time_frame>From start of participation of the patient to end of the treatment period (up to 8 weeks)</time_frame>
    <description>Blood test results (hepatic assessment) at the start and at the end of the study</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Blood test results (blood cells count) at the start and at the end of the study</measure>
    <time_frame>From start of participation of the patient to end of the treatment period (up to 8 weeks)</time_frame>
    <description>Blood test results (blood cells count) at the start and at the end of the study</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Frequency and nature of the adverse events throughout the study</measure>
    <time_frame>From start of participation of the patient to end of the treatment period (up to 8 weeks)</time_frame>
    <description>Frequency and nature of the adverse events throughout the study</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">15</enrollment>
  <condition>Primary Hyperoxaluria</condition>
  <arm_group>
    <arm_group_label>stiripentol (Diacomit)</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>stiripentol (Diacomit)</intervention_name>
    <description>Administration of stiripentol per os</description>
    <arm_group_label>stiripentol (Diacomit)</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Patient with primary hyperoxaluria type 1, 2 or 3, diagnosed according to standard&#xD;
             methods&#xD;
&#xD;
          -  Having at least one molar ratio [oxaluria / creatinuria] greater than 0.08 since&#xD;
             diagnosis&#xD;
&#xD;
          -  Having Glomerular Filtration Rate ≥ 45 mL / min / 1.73m2&#xD;
&#xD;
          -  Age ≥ 6 months&#xD;
&#xD;
          -  Having read, or whose parents have read, the information note and signed the consent&#xD;
             form. For children, if their level of understanding allows, their assent will also be&#xD;
             sought&#xD;
&#xD;
          -  Proficient enough, or whose parents or legal representatives have sufficient mastery,&#xD;
             the French language to read, understand and complete study documents&#xD;
&#xD;
          -  Affiliate or beneficiary of a social security scheme&#xD;
&#xD;
          -  Ability to respect the protocol, including treatment, and can be followed regularly in&#xD;
             the study&#xD;
&#xD;
          -  For pubertal patients, contraception deemed effective by the investigator or&#xD;
             abstinence&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Introduction, discontinuation or dose modification of vitamin B6 or potassium citrate&#xD;
             treatment within 4 weeks prior to the inclusion visit&#xD;
&#xD;
          -  Consumption of jelly candies and / or dark chocolate in the week preceding the study&#xD;
&#xD;
          -  Patient having a kidney and / or liver transplant&#xD;
&#xD;
          -  Presence of a clinically significant acute or chronic pathology, other than primary&#xD;
             hyperoxaluria, that may interfere with the evaluation of the study results according&#xD;
             to the investigator&#xD;
&#xD;
          -  During biological or physical examinations, presence of significant anomaly (s)&#xD;
             inconsistent with participation in the study according to the investigator&#xD;
&#xD;
          -  History of severe allergy, asthma, skin rash or hypersensitivity to a drug&#xD;
&#xD;
          -  Treatment affecting hepatic metabolism (cimetidine, ketoconazole, fluconazole,&#xD;
             itraconazole, phenytoin, rifampicin, rifabutin) in progress or taken during the month&#xD;
             preceding the start of the study&#xD;
&#xD;
          -  Treatment affecting the renal tubule (probenecid, β-lactams, ...) in progress or taken&#xD;
             during the last two weeks preceding the start of the study&#xD;
&#xD;
          -  Presence of a pathology or treatment that, according to the investigator, renders the&#xD;
             subject unfit&#xD;
&#xD;
          -  Contraindications to stiripentol as defined in the current SmPC (hypersensitivity to&#xD;
             the active substance or to any of the excipients listed in section 6.1 of the SmPC,&#xD;
             history of psychosis in the form of delusional episodes)&#xD;
&#xD;
          -  Pregnant or lactating woman&#xD;
&#xD;
          -  Patient under guardianship&#xD;
&#xD;
          -  Patient concurrently participating in another clinical trial or exclusion period&#xD;
             following a previous trial&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Months</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Hôpital Necker</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hôpital Robert Debré</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hôpital Tenon</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>November 2020</verification_date>
  <study_first_submitted>January 18, 2019</study_first_submitted>
  <study_first_submitted_qc>January 25, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 28, 2019</study_first_posted>
  <last_update_submitted>March 25, 2021</last_update_submitted>
  <last_update_submitted_qc>March 25, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">March 26, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hyperoxaluria, Primary</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Stiripentol</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- 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/NCT03819647
org study id: HYPOP (STP194)
nct id: NCT03819647
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
is us export: No
Evaluation of the efficacy of stiripentol (Diacomit) as monotherapy for the treatment of
      primary hyperoxaluria.

      Pilot clinical study, open, prospective and multicenter.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Relative variation (%) of the molar ratio [oxaluria / creatinuria] between baseline and after two weeks of treatment.
time frame: Change (%) of the molar ratio [oxaluria / creatinuria] between the baseline value (average of 3 measures done during pre-treatment period) and the value (average of 2 measures) after 2 weeks of treatment.
description: Relative variation (%) of the molar ratio [oxaluria / creatinuria] between baseline and after two weeks of treatment.
measure: Relative variation (%) of the molar ratio [oxaluria / creatinuria] between baseline and after three weeks of treatment.
time frame: Change (%) of the molar ratio [oxaluria / creatinuria] between the baseline value (average of 3 measures done during pre-treatment period) and the value (average of 2 measures) after 3 weeks of treatment.
description: Relative variation (%) of the molar ratio [oxaluria / creatinuria] between baseline and after three weeks of treatment.
measure: Response to treatment defined by a decrease> 20% of the molar ratio [oxaluria / creatinuria]
time frame: 3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)
description: Response to treatment defined by a decrease> 20% of the molar ratio [oxaluria / creatinuria]
measure: Relative variation (%) of supersaturation of urine with calcium oxalate between the start and the end of treatment period
time frame: 3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)
description: Relative variation (%) of supersaturation of urine with calcium oxalate between the start and the end of treatment period
measure: Relative variation (%) in overall crystalline volume measured by crystalluria on fresh urine between the start and the end of treatment period
time frame: 3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)
description: Relative variation (%) in overall crystalline volume measured by crystalluria on fresh urine between the start and the end of treatment period
measure: Effect of stiripentol dose increase on absolute decrease of the molar ratio [oxaluria / creatinuria]
time frame: 3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)
description: Effect of stiripentol dose increase on absolute decrease of the molar ratio [oxaluria / creatinuria]
measure: Effect of stiripentol dose increase on relative decrease (%) of the molar ratio [oxaluria / creatinuria]
time frame: 3 measures from inclusion to first treatment intake (= baseline value), then 2 measures at Day 14 and Day 15 respectively (=value after 2 weeks of treatment), and 2 measures at Day 20 and Day 21 respectively (=value after 3 weeks of treatment)
description: Effect of stiripentol dose increase on relative decrease (%) of the molar ratio [oxaluria / creatinuria]
measure: Blood test results (hepatic assessment) at the start and at the end of the study
time frame: From start of participation of the patient to end of the treatment period (up to 8 weeks)
description: Blood test results (hepatic assessment) at the start and at the end of the study
measure: Blood test results (blood cells count) at the start and at the end of the study
time frame: From start of participation of the patient to end of the treatment period (up to 8 weeks)
description: Blood test results (blood cells count) at the start and at the end of the study
measure: Frequency and nature of the adverse events throughout the study
time frame: From start of participation of the patient to end of the treatment period (up to 8 weeks)
description: Frequency and nature of the adverse events throughout the study
arm group label: stiripentol (Diacomit)
arm group type: Experimental
intervention type: Drug
intervention name: stiripentol (Diacomit)
description: Administration of stiripentol per os
arm group label: stiripentol (Diacomit)
criteria: 
gender: All
minimum age: 6 Months
maximum age: N/A
healthy volunteers: No
facility: 
facility: 
facility: 
country: France
responsible party type: Sponsor
mesh term: Hyperoxaluria, Primary
mesh term: Stiripentol
sharing ipd: No