Fatty Acid Oxidation Disorders
Conditions
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.
Interventions
DRUGLow Dose REN001
Oral
DRUGHigh Dose REN001
Oral
Sponsors
Reneo Pharma Ltd
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse Events | Continous to Week 12 | Number of participants with Adverse Events (AEs) as a measure of safety and tolerability |
Countries
France, Spain, United States
Outcome results
None listed