A Phase 1b Study of the Safety of REN001 in Patients With Fatty Acid Oxidation Disorders

Phase 1

Completed

• Conditions: Fatty Acid Oxidation Disorders
• Interventions: Drug: High Dose REN001; Drug: Low Dose REN001

• Registration Number: NCT03833128
• Lead Sponsor: Reneo Pharma Ltd

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 Timeline

• Study First Submitted: 2019-02-04
• Study First Submitted QC: 2019-02-05
• Study First Posted: 2019-02-06
• Study Start: 2019-04-04
• Primary Completion: 2022-01-24
• Study Completion: 2022-03-21
• Status Verified: 2022-12
• Last Update Submitted: 2022-12-07
• Last Update Posted: 2022-12-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 24

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Group 3 - Part B	High Dose REN001	REN001 High Dose oral once daily x 12 weeks
Group 1 - Part A	Low Dose REN001	REN001 Low Dose oral once daily x 12 weeks
Group 2 - Part A	High Dose REN001	REN001 High Dose oral once daily x 12 weeks

Primary Outcome Measures

Name	Time	Method
Adverse Events	Continous to Week 12	Number of participants with Adverse Events (AEs) as a measure of safety and tolerability

Trial Locations

Locations (7)

Division of Medical Genetics, University Utah; 🇺🇸 Salt Lake City, Utah, United States

UT Southwestern Medical Center; 🇺🇸 Dallas, Texas, United States

Neurology department, Raymond-Poincaré Teaching Hospital, Nord/Est/Ile de France Neuromuscular Reference Center; 🇫🇷 Garches, France

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Oregon Health and Science University; 🇺🇸 Portland, Oregon, United States

Servicio de Neurología - Unidad de Neuromuscular Centro de Referencia Nacional de Enfermedades Neuromusculares raras Instituto de Investigación i+12; 🇪🇸 Madrid, Spain

Children's Hospital of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States