A Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of 24 Weeks Treatment With REN001 in Patients With Primary Mitochondrial Myopathy

Reneo Pharma Ltd41 sites in 15 countries213 target enrollmentMay 21, 2021

ConditionsPrimary Mitochondrial Myopathy

InterventionsMavodelpar Placebo

Overview

Phase: Phase 2
Intervention: Mavodelpar
Conditions: Primary Mitochondrial Myopathy
Sponsor: Reneo Pharma Ltd
Enrollment: 213
Locations: 41
Primary Endpoint: Change in Distance Walked During a 12 Minute Walk Test
Status: Completed
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This is a randomized, double-blind, placebo-controlled, parallel group, multi-centre, study designed to investigate the efficacy and safety of REN001 administered once daily over a 24-week period to patients with PMM.

Registry

clinicaltrials.gov

Start Date

May 21, 2021

End Date

October 5, 2023

Last Updated

last year

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Reneo Pharma Ltd

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Subjects age 18 years or older with PMM as defined by the International Workshop: Outcome measures and clinical trial readiness in primary mitochondrial myopathies in children and adult (Mancuso et al 2017).
•A confirmed PMM diagnosis due to known pathogenic gene mutation or deletion of the mitochondrial genome. The Sponsor may authorize local genetic testing at Screening, if required, but results must be available prior to randomization of the subject.
•Documented PMM primarily characterized by exercise intolerance or active muscle pain.
•Subjects must be ambulatory and able to perform the walking tests independently (walking aids are allowed).
•Have no changes to any therapeutic exercise regimen within 30 days prior to Day 1 and be willing to remain on the same therapeutic exercise regimen for the duration of the study.
•Females should be either of non-child-bearing potential or must agree to use highly effective methods of contraception from Screening through to 30 days after last dose in the study. Males with partners who are WOCBP must also use contraception.
•Concomitant medications (including supplements) must be stable for at least 1 month prior to enrolment and throughout participation in the study.
•Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
•Participation in a prior REN001 (previously known as HPP-593) study.
•Currently taking or anticipated to need a PPAR agonist during the study.