<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/NCT02201368</url>
  </required_header>
  <id_info>
    <org_study_id>2007/084</org_study_id>
    <secondary_id>2007-005578-29</secondary_id>
    <nct_id>NCT02201368</nct_id>
  </id_info>
  <brief_title>Triheptanoin Treatment Trial for Patients With Long-chain Fatty Acid Beta-oxidation Defects</brief_title>
  <official_title>Study to Evaluate the Effectiveness of Dietary Treatment With Triheptanoin in Patients With Long-chain Fatty Acid Beta-oxidation Defects</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Maria Luz Couce Pico</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Fundación Ramón Domínguez</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Hospital Clinico Universitario de Santiago</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to determine if administration Triheptanoin is an effective&#xD;
      treatment for defects of the long-chain fatty acid beta-oxidation in young adults or adults.&#xD;
      Period of treatment and follow-up will be 16 months.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Triheptanoin treatment, in patients with long-chain fatty acid beta-oxidation defects, could&#xD;
      cause not only a great improvement in their quality of life, also could prevent&#xD;
      life-threatening signs, reducing symptoms and serious complications of their disease, like&#xD;
      cardiomyopathy, Reye-like syndrome episodes and rhabdomyolysis. This result would occur by&#xD;
      the effect of propionyl CoA primer on the Krebs cycle and, at the same time, would produce a&#xD;
      gluconeogenic effect.&#xD;
&#xD;
      This treatment opens the door to be used in other diseases such as pyruvate carboxylase&#xD;
      deficiency, glycogen storage disease and other diseases with energy problems.&#xD;
&#xD;
      All patients will be followed up until 16 months.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Withdrawn</overall_status>
  <why_stopped>
    IMP management difficulties&#xD;
  </why_stopped>
  <start_date>November 2009</start_date>
  <completion_date type="Actual">October 2011</completion_date>
  <primary_completion_date type="Actual">March 2010</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>Crossover Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of metabolic decompensation.</measure>
    <time_frame>up to 16 months</time_frame>
    <description>This is a combined endpoint, including the number and/or severity of episodes of hypoglycemia, rhabdomyolysis, cardiomyopathy and liver failure after starting treatment with trihepatnoin.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Differences in the profiles of acylcarnitines with control.</measure>
    <time_frame>6 months and 6 months in each arm treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Average values of transaminase and creatin kinase.</measure>
    <time_frame>6 months and 6 months in each arm treatment</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Differences in the fatty acid composition of plasma and red blood cells.</measure>
    <time_frame>6 months and 6 months in each arm treatment</time_frame>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">0</enrollment>
  <condition>Long-chain Fatty Acid Transport Deficiency</condition>
  <arm_group>
    <arm_group_label>Triheptanoin</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>MCT (Medium-Chain Triglycerides)</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Triheptanoin (SpezialölÒ 107®)</intervention_name>
    <description>Randomization: Treatment with Triheptanoin for 6 months&#xD;
Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months&#xD;
Crossover: Treatment with MCT(Medium-Chain Triglycerides)for 6 months</description>
    <arm_group_label>MCT (Medium-Chain Triglycerides)</arm_group_label>
    <arm_group_label>Triheptanoin</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Dietary Supplement</intervention_type>
    <intervention_name>MCT (Medium-Chain Triglycerides)</intervention_name>
    <description>Randomization: Treatment with MCT(Medium-Chain Triglycerides)for 6 months&#xD;
Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months&#xD;
Crossover: Treatment with Triheptanoin for 6 months</description>
    <arm_group_label>MCT (Medium-Chain Triglycerides)</arm_group_label>
    <arm_group_label>Triheptanoin</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        All patients with any of the following conditions:&#xD;
&#xD;
          -  Long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency (LCHAD).&#xD;
&#xD;
          -  Very long-chain acyl-coenzyme A dehydrogenase deficiency (VLCAD.)&#xD;
&#xD;
          -  Mitochondrial trifunctional protein (MTP).&#xD;
&#xD;
          -  Carnitine palmitoyltransferase I deficiency (CPT I).&#xD;
&#xD;
          -  Carnitine Palmitoyltransferase II (CPT II).&#xD;
&#xD;
          -  Carnitine-acylcarnitine translocase deficiency (CACT).&#xD;
&#xD;
        Positive skin biopsy: patients were deemed to commence the dietary treatment with&#xD;
        Triheptanoin after evaluating the individual response in vitro in cultured fibroblasts.&#xD;
        This response is based on the measurement of the production of propionyl-CoA, the&#xD;
        incubation with fatty acids odd-chain, compared with control group fibroblasts.&#xD;
&#xD;
        The informed consent must be signed by the patient or family, in the case of minors.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  No patient/family collaboration or the application of dietary treatment.&#xD;
&#xD;
          -  No in vitro test response.&#xD;
&#xD;
          -  Do not meet the inclusion criteria.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Mª Luz Couce Pico</last_name>
    <role>Study Chair</role>
    <affiliation>Hospital Clínico Universitario de Santiago</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Jaume Campistol Plana</last_name>
    <role>Principal Investigator</role>
    <affiliation>Hospital Sant Joan de Déu. Barcelona</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Mercedes Martínez-Pardo</last_name>
    <role>Principal Investigator</role>
    <affiliation>Hospital Universitario Ramon y Cajal</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Mónica Ruiz Pons</last_name>
    <role>Principal Investigator</role>
    <affiliation>Hospital Clínico Universitario Ntra. Sra. de Candelaria. Tenerife</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Mª Teresa García Silva</last_name>
    <role>Principal Investigator</role>
    <affiliation>Hospital 12 de Octubre. Madrid</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Pablo Sanjurjo</last_name>
    <role>Principal Investigator</role>
    <affiliation>Hospital de Cruces de Baracaldo - Bilbao</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Koldo Aldamiz</last_name>
    <role>Principal Investigator</role>
    <affiliation>Hospital de Cruces de Baracaldo - Bilbao</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Inmaculada García Jiménez</last_name>
    <role>Principal Investigator</role>
    <affiliation>Hospital Universitario Miguel Servet, Zaragoza</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Hospital Clínico Universitario de Santiago</name>
      <address>
        <city>Santiago de Compostela</city>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Spain</country>
  </location_countries>
  <verification_date>July 2014</verification_date>
  <study_first_submitted>March 22, 2013</study_first_submitted>
  <study_first_submitted_qc>July 25, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">July 28, 2014</study_first_posted>
  <last_update_submitted>July 25, 2014</last_update_submitted>
  <last_update_submitted_qc>July 25, 2014</last_update_submitted_qc>
  <last_update_posted type="Estimate">July 28, 2014</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Hospital Clinico Universitario de Santiago</investigator_affiliation>
    <investigator_full_name>Maria Luz Couce Pico</investigator_full_name>
    <investigator_title>MD</investigator_title>
  </responsible_party>
  <keyword>Triglycerides; Lipid Metabolism; Fatty Acids.</keyword>
  <!-- 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/NCT02201368
org study id: 2007/084
secondary id: 2007-005578-29
nct id: NCT02201368
lead sponsor: 
collaborator: 
has dmc: No
The purpose of this study is to determine if administration Triheptanoin is an effective
      treatment for defects of the long-chain fatty acid beta-oxidation in young adults or adults.
      Period of treatment and follow-up will be 16 months.
Triheptanoin treatment, in patients with long-chain fatty acid beta-oxidation defects, could
      cause not only a great improvement in their quality of life, also could prevent
      life-threatening signs, reducing symptoms and serious complications of their disease, like
      cardiomyopathy, Reye-like syndrome episodes and rhabdomyolysis. This result would occur by
      the effect of propionyl CoA primer on the Krebs cycle and, at the same time, would produce a
      gluconeogenic effect.

      This treatment opens the door to be used in other diseases such as pyruvate carboxylase
      deficiency, glycogen storage disease and other diseases with energy problems.

      All patients will be followed up until 16 months.
allocation: Randomized
intervention model: Crossover Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of metabolic decompensation.
time frame: up to 16 months
description: This is a combined endpoint, including the number and/or severity of episodes of hypoglycemia, rhabdomyolysis, cardiomyopathy and liver failure after starting treatment with trihepatnoin.
measure: Differences in the profiles of acylcarnitines with control.
time frame: 6 months and 6 months in each arm treatment
measure: Average values of transaminase and creatin kinase.
time frame: 6 months and 6 months in each arm treatment
measure: Differences in the fatty acid composition of plasma and red blood cells.
time frame: 6 months and 6 months in each arm treatment
arm group label: Triheptanoin
arm group type: Experimental
arm group label: MCT (Medium-Chain Triglycerides)
arm group type: Active Comparator
intervention type: Drug
intervention name: Triheptanoin (SpezialölÒ 107®)
description: Randomization: Treatment with Triheptanoin for 6 months
Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months
Crossover: Treatment with MCT(Medium-Chain Triglycerides)for 6 months
arm group label: MCT (Medium-Chain Triglycerides)
arm group label: Triheptanoin
intervention type: Dietary Supplement
intervention name: MCT (Medium-Chain Triglycerides)
description: Randomization: Treatment with MCT(Medium-Chain Triglycerides)for 6 months
Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months
Crossover: Treatment with Triheptanoin for 6 months
arm group label: MCT (Medium-Chain Triglycerides)
arm group label: Triheptanoin
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Mª Luz Couce Pico
role: Study Chair
affiliation: Hospital Clínico Universitario de Santiago
last name: Jaume Campistol Plana
role: Principal Investigator
affiliation: Hospital Sant Joan de Déu. Barcelona
last name: Mercedes Martínez-Pardo
role: Principal Investigator
affiliation: Hospital Universitario Ramon y Cajal
last name: Mónica Ruiz Pons
role: Principal Investigator
affiliation: Hospital Clínico Universitario Ntra. Sra. de Candelaria. Tenerife
last name: Mª Teresa García Silva
role: Principal Investigator
affiliation: Hospital 12 de Octubre. Madrid
last name: Pablo Sanjurjo
role: Principal Investigator
affiliation: Hospital de Cruces de Baracaldo - Bilbao
last name: Koldo Aldamiz
role: Principal Investigator
affiliation: Hospital de Cruces de Baracaldo - Bilbao
last name: Inmaculada García Jiménez
role: Principal Investigator
affiliation: Hospital Universitario Miguel Servet, Zaragoza
facility: 
country: Spain
responsible party type: Sponsor-Investigator
investigator affiliation: Hospital Clinico Universitario de Santiago
investigator full name: Maria Luz Couce Pico
investigator title: MD