A Phase 1b Study of the Safety of REN001 in Patients With Fatty Acid Oxidation Disorders
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)
1 other identifier
interventional
24
3 countries
7
Brief Summary
The purpose of this Phase 1b study is to assess REN001 safety in subjects with fatty acid oxidation disorders.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Apr 2019
Typical duration for phase_1
7 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 4, 2019
CompletedFirst Posted
Study publicly available on registry
February 6, 2019
CompletedStudy Start
First participant enrolled
April 4, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 24, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
March 21, 2022
CompletedDecember 9, 2022
December 1, 2022
2.8 years
February 4, 2019
December 7, 2022
Conditions
Outcome Measures
Primary Outcomes (1)
Adverse Events
Number of participants with Adverse Events (AEs) as a measure of safety and tolerability
Continous to Week 12
Study Arms (3)
Group 1 - Part A
EXPERIMENTALREN001 Low Dose oral once daily x 12 weeks
Group 2 - Part A
EXPERIMENTALREN001 High Dose oral once daily x 12 weeks
Group 3 - Part B
EXPERIMENTALREN001 High Dose oral once daily x 12 weeks
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Reneo Pharma Ltdlead
Study Sites (7)
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
UT Southwestern Medical Center
Dallas, Texas, 75390-8591, United States
Division of Medical Genetics, University Utah
Salt Lake City, Utah, 84108, United States
Neurology department, Raymond-Poincaré Teaching Hospital, Nord/Est/Ile de France Neuromuscular Reference Center
Garches, 92380, France
Servicio de Neurología - Unidad de Neuromuscular Centro de Referencia Nacional de Enfermedades Neuromusculares raras Instituto de Investigación i+12
Madrid, CP 28041, Spain
Study Officials
- PRINCIPAL INVESTIGATOR
Jerry Vockley, M.D., Ph.D.
University of Pittsburgh Medical Center
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 4, 2019
First Posted
February 6, 2019
Study Start
April 4, 2019
Primary Completion
January 24, 2022
Study Completion
March 21, 2022
Last Updated
December 9, 2022
Record last verified: 2022-12