<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/NCT03833128</url>
  </required_header>
  <id_info>
    <org_study_id>REN001-102</org_study_id>
    <nct_id>NCT03833128</nct_id>
  </id_info>
  <brief_title>A Phase 1b Study of the Safety of REN001 in Patients With Fatty Acid Oxidation Disorders</brief_title>
  <official_title>An Open Label Study to Determine the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Subjects With Fatty Acid Oxidation Disorders (FAOD)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Reneo Pharma Ltd</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Reneo Pharma Ltd</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>
      The purpose of this Phase 1b study is to assess REN001 safety in subjects with fatty acid&#xD;
      oxidation disorders.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a Phase 1b, open-label, multiple-dose study of the safety and tolerability of 2 dose&#xD;
      levels of REN001 in subjects with fatty acid oxidation disorders (FAODs) with confirmed&#xD;
      mutations in the Carnitine palmitoyltransferase II deficiency (CPT2), Very long-chain&#xD;
      Acyl-CoA dehydrogenase deficiency (VLCAD), Long-chain 3-hydroxyacyl-CoA dehydrogenase&#xD;
      deficiency (LCHAD) or Trifunctional Protein Deficiency (TFP). All subjects will provide&#xD;
      written consent prior to commencing any study related activities or assessments. Potential&#xD;
      subjects will be screened for study participation up to 8 weeks prior to the start of&#xD;
      dosing.The study is divided into two parts, Part A and Part B. Part A has finished enrollment&#xD;
      and further eligible patients will participate in Part B only.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">April 4, 2019</start_date>
  <completion_date type="Actual">March 21, 2022</completion_date>
  <primary_completion_date type="Actual">January 24, 2022</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Sequential Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Adverse Events</measure>
    <time_frame>Continous to Week 12</time_frame>
    <description>Number of participants with Adverse Events (AEs) as a measure of safety and tolerability</description>
  </primary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">24</enrollment>
  <condition>Fatty Acid Oxidation Disorders</condition>
  <arm_group>
    <arm_group_label>Group 1 - Part A</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>REN001 Low Dose oral once daily x 12 weeks</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Group 2 - Part A</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>REN001 High Dose oral once daily x 12 weeks</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Group 3 - Part B</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>REN001 High Dose oral once daily x 12 weeks</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Low Dose REN001</intervention_name>
    <description>Oral</description>
    <arm_group_label>Group 1 - Part A</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>High Dose REN001</intervention_name>
    <description>Oral</description>
    <arm_group_label>Group 2 - Part A</arm_group_label>
    <arm_group_label>Group 3 - Part B</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        Subjects must give written, signed and dated informed consent&#xD;
&#xD;
        Confirmed diagnosis of FAOD&#xD;
&#xD;
        A diagnostic acylcarnitine profile, in blood or cultured fibroblasts&#xD;
&#xD;
        A stable treatment regimen for at least 30 days prior to enrollment&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        Unstable or poorly controlled disease&#xD;
&#xD;
        Treatment with an investigational drug within 1 month or within 5 half-lives, whichever is&#xD;
        longer&#xD;
&#xD;
        Have been hospitalized within 3 months prior to screening for any major medical event&#xD;
&#xD;
        Pregnant or nursing females&#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>Jerry Vockley, M.D., Ph.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Pittsburgh Medical Center</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Children's Hospital Colorado</name>
      <address>
        <city>Aurora</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oregon Health and Science University</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97239</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Pittsburgh</name>
      <address>
        <city>Pittsburgh</city>
        <state>Pennsylvania</state>
        <zip>15224</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UT Southwestern Medical Center</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75390-8591</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Division of Medical Genetics, University Utah</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84108</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Neurology department, Raymond-Poincaré Teaching Hospital, Nord/Est/Ile de France Neuromuscular Reference Center</name>
      <address>
        <city>Garches</city>
        <zip>92380</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Servicio de Neurología - Unidad de Neuromuscular Centro de Referencia Nacional de Enfermedades Neuromusculares raras Instituto de Investigación i+12</name>
      <address>
        <city>Madrid</city>
        <zip>CP 28041</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
    <country>Spain</country>
    <country>United States</country>
  </location_countries>
  <verification_date>December 2022</verification_date>
  <study_first_submitted>February 4, 2019</study_first_submitted>
  <study_first_submitted_qc>February 5, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">February 6, 2019</study_first_posted>
  <last_update_submitted>December 7, 2022</last_update_submitted>
  <last_update_submitted_qc>December 7, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">December 9, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <!-- 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/NCT03833128
org study id: REN001-102
nct id: NCT03833128
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this Phase 1b study is to assess REN001 safety in subjects with fatty acid
      oxidation disorders.
This is a Phase 1b, open-label, multiple-dose study of the safety and tolerability of 2 dose
      levels of REN001 in subjects with fatty acid oxidation disorders (FAODs) with confirmed
      mutations in the Carnitine palmitoyltransferase II deficiency (CPT2), Very long-chain
      Acyl-CoA dehydrogenase deficiency (VLCAD), Long-chain 3-hydroxyacyl-CoA dehydrogenase
      deficiency (LCHAD) or Trifunctional Protein Deficiency (TFP). All subjects will provide
      written consent prior to commencing any study related activities or assessments. Potential
      subjects will be screened for study participation up to 8 weeks prior to the start of
      dosing.The study is divided into two parts, Part A and Part B. Part A has finished enrollment
      and further eligible patients will participate in Part B only.
allocation: Non-Randomized
intervention model: Sequential Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Adverse Events
time frame: Continous to Week 12
description: Number of participants with Adverse Events (AEs) as a measure of safety and tolerability
arm group label: Group 1 - Part A
arm group type: Experimental
description: REN001 Low Dose oral once daily x 12 weeks
arm group label: Group 2 - Part A
arm group type: Experimental
description: REN001 High Dose oral once daily x 12 weeks
arm group label: Group 3 - Part B
arm group type: Experimental
description: REN001 High Dose oral once daily x 12 weeks
intervention type: Drug
intervention name: Low Dose REN001
description: Oral
arm group label: Group 1 - Part A
intervention type: Drug
intervention name: High Dose REN001
description: Oral
arm group label: Group 2 - Part A
arm group label: Group 3 - Part B
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Jerry Vockley, M.D., Ph.D.
role: Principal Investigator
affiliation: University of Pittsburgh Medical Center
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: France
country: Spain
country: United States
responsible party type: Sponsor