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)
Trial Snapshot
- Phase
- Phase 1
- Status
- Completed
- Sponsor
- Reneo Pharma Ltd
- Enrollment
- 24
- Locations
- 7
- Primary Endpoint
- Adverse Events
Study Overview
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 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
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
Group 1 - Part A
REN001 Low Dose oral once daily x 12 weeks
Intervention: Low Dose REN001 (Drug)
Group 2 - Part A
REN001 High Dose oral once daily x 12 weeks
Intervention: High Dose REN001 (Drug)
Group 3 - Part B
REN001 High Dose oral once daily x 12 weeks
Intervention: High Dose REN001 (Drug)
Outcomes
Primary Outcomes
Adverse Events
Time Frame: Continous to Week 12
Number of participants with Adverse Events (AEs) as a measure of safety and tolerability
Secondary Outcomes
No secondary outcomes reported