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

Fatty Acid Oxidation Disorders Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03833128
• Other study ID #: REN001-102
• Status: Completed
• Phase: Phase 1
• Start date: April 4, 2019
• Est. completion date: March 21, 2022

Study information

• Verified date: December 2022
• Source: Reneo Pharma Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this Phase 1b study is to assess REN001 safety in subjects with fatty acid oxidation disorders.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: March 21, 2022
• Est. primary completion date: January 24, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Disease
• Fatty Acid Oxidation Disorders

Intervention

Drug:
• Low Dose REN001
Oral
• High Dose REN001
Oral

Locations

Country	Name	City	State
France	Neurology department, Raymond-Poincaré Teaching Hospital, Nord/Est/Ile de France Neuromuscular Reference Center	Garches
Spain	Servicio de Neurología - Unidad de Neuromuscular Centro de Referencia Nacional de Enfermedades Neuromusculares raras Instituto de Investigación i+12	Madrid
United States	Children's Hospital Colorado	Aurora	Colorado
United States	UT Southwestern Medical Center	Dallas	Texas
United States	Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon
United States	Division of Medical Genetics, University Utah	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Reneo Pharma Ltd

Countries where clinical trial is conducted

United States,  France,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse Events	Number of participants with Adverse Events (AEs) as a measure of safety and tolerability	Continous to Week 12