NCT03833128

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)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Reneo Pharma Ltd · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Fatty Acid Oxidation Disorders

Interventions

Type	Name	Description
DRUG	Low Dose REN001	Oral
DRUG	High Dose REN001	Oral

Timeline

• Start date: 2019-04-04
• Primary completion: 2022-01-24
• Completion: 2022-03-21
• First posted: 2019-02-06
• Last updated: 2022-12-09

Locations

7 sites across 3 countries: United States, France, Spain

Regulatory

• FDA-regulated drug study