Status:
COMPLETED
A Phase 1b Study of the Safety of REN001 in Patients With Fatty Acid Oxidation Disorders
Lead Sponsor:
Reneo Pharma Ltd
Conditions:
Fatty Acid Oxidation Disorders
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief Summary
The purpose of this Phase 1b study is to assess REN001 safety in subjects with fatty acid oxidation disorders.
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 Ca...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
April 4 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 21 2022
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT03833128
Start Date
April 4 2019
End Date
March 21 2022
Last Update
December 9 2022
Active Locations (7)
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1
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
2
Oregon Health and Science University
Portland, Oregon, United States, 97239
3
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
4
UT Southwestern Medical Center
Dallas, Texas, United States, 75390-8591