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A Phase 1b Study of the Safety of REN001 in Patients With Fatty Acid Oxidation Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03833128
Recruitment Status : Completed
First Posted : February 6, 2019
Last Update Posted : December 9, 2022
Sponsor:
Information provided by (Responsible Party):
Reneo Pharma Ltd

Study Description
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Brief Summary:
The purpose of this Phase 1b study is to assess REN001 safety in subjects with fatty acid oxidation disorders.

Condition or disease Intervention/treatment Phase
Fatty Acid Oxidation Disorders Drug: Low Dose REN001 Drug: High Dose REN001 Phase 1

Detailed Description:
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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : April 4, 2019
Actual Primary Completion Date : January 24, 2022
Actual Study Completion Date : March 21, 2022
Arms and Interventions
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Arm Intervention/treatment
Experimental: Group 1 - Part A
REN001 Low Dose oral once daily x 12 weeks
 Drug: Low Dose REN001
Oral
Experimental: Group 2 - Part A
REN001 High Dose oral once daily x 12 weeks
 Drug: High Dose REN001
Oral
Experimental: Group 3 - Part B
REN001 High Dose oral once daily x 12 weeks
 Drug: High Dose REN001
Oral


Outcome Measures
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Primary Outcome Measures :
  1. Adverse Events [ Time Frame: Continous to Week 12 ]
    Number of participants with Adverse Events (AEs) as a measure of safety and tolerability


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Subjects must give written, signed and dated informed consent

Confirmed diagnosis of FAOD

A diagnostic acylcarnitine profile, in blood or cultured fibroblasts

A stable treatment regimen for at least 30 days prior to enrollment

Exclusion Criteria:

Unstable or poorly controlled disease

Treatment with an investigational drug within 1 month or within 5 half-lives, whichever is longer

Have been hospitalized within 3 months prior to screening for any major medical event

Pregnant or nursing females

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03833128


Locations
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United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
UT Southwestern Medical Center
Dallas, Texas, United States, 75390-8591
United States, Utah
Division of Medical Genetics, University Utah
Salt Lake City, Utah, United States, 84108
France
Neurology department, Raymond-Poincaré Teaching Hospital, Nord/Est/Ile de France Neuromuscular Reference Center
Garches, France, 92380
Spain
Servicio de Neurología - Unidad de Neuromuscular Centro de Referencia Nacional de Enfermedades Neuromusculares raras Instituto de Investigación i+12
Madrid, Spain, CP 28041
Sponsors and Collaborators
Reneo Pharma Ltd
Investigators
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Principal Investigator: Jerry Vockley, M.D., Ph.D. University of Pittsburgh Medical Center
More Information
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Responsible Party: Reneo Pharma Ltd
ClinicalTrials.gov Identifier: NCT03833128    
Other Study ID Numbers: REN001-102
First Posted: February 6, 2019    Key Record Dates
Last Update Posted: December 9, 2022
Last Verified: December 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Disease
Pathologic Processes