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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04535609
Other study ID # REN001-201
Secondary ID
Status Completed
Phase Phase 2
First received
Last updated
Start date May 21, 2021
Est. completion date October 5, 2023

Study information

Verified date April 2024
Source Reneo Pharma Ltd
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Completed
Enrollment 213
Est. completion date October 5, 2023
Est. primary completion date September 12, 2023
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: 1. 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). 2. 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. 3. Documented PMM primarily characterized by exercise intolerance or active muscle pain. 4. Subjects must be ambulatory and able to perform the walking tests independently (walking aids are allowed). 5. 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. 6. 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. 7. Concomitant medications (including supplements) must be stable for at least 1 month prior to enrolment and throughout participation in the study. 8. Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study. Exclusion: 1. Participation in a prior REN001 (previously known as HPP-593) study. 2. Currently taking or anticipated to need a PPAR agonist during the study. 3. Subjects with bone deformities or motor abnormalities other than related to the mitochondrial myopathy that may interfere with the outcome measures. 4. Clinically significant kidney disease or impairment calculated as eGFR Grade 2 or above <60ml/min/1.73m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation at Screening. 5. Clinically significant liver disease or impairment of AST or ALT Grade 2 or above (>2.5 x ULN), or Total bilirubin > 1.6 x ULN or >ULN with other signs and symptoms of hepatotoxicity at Screening. 6. Subjects with uncontrolled diabetes and/or a Screening HbA1c of =11%. 7. Evidence of significant concomitant clinical disease that may need a change in management during the study or could interfere with the conduct or safety of this study. (Stable well-controlled chronic conditions such hypercholesterolemia, gastroesophageal reflux, or depression under control with medication (other than tricyclic antidepressants), are acceptable provided the symptoms and medications would not be predicted to compromise safety or interfere with the tests and interpretations of this study.) 8. Subjects with a history of cancer. A history of in situ basal cell carcinoma in the skin is allowed. 9. Clinically significant cardiac disease and/or clinically significant ECG abnormalities such as 2nd degree heart block, symptomatic tachyarrhythmia or unstable arrythmia (right bundle branch block, left fascicular block and long PR interval are not excluded) that in the opinion of the Investigator should exclude the subject from completing exercise tests. 10. Evidence of hospitalization for rhabdomyolysis within the year prior to enrolment. 11. Pregnant or nursing females. 12. History of sensitivity to PPAR agonists.

Study Design


Intervention

Drug:
Mavodelpar
Once daily
Placebo
Once daily

Locations

Country Name City State
Australia PARC Clinical Research Adelaide South Australia
Australia The Alfred Hospital Melbourne Victoria
Australia Royal North Shore Hospital St. Leonards New South Wales
Belgium University Hospital Leuven Leuven
Canada M.A.G.I.C. Clinic (Metabolics and Genetics in Calgary) Calgary Alberta
Canada Adult Metabolic Diseases Clinic, Vancouver General Hospital Vancouver British Columbia
Czechia General University Hospital in Prague Prague
Denmark Rigshospitalet, University of Copenhagen Copenhagen
France Centre Hospitalier Universitaire d' Angers Angers Pays De La Loire
France Hôpital Neurologique Pierre Wertheimer Bron
France Hôpital Roger Salengro Lille Hauts De France
France CHU de Nice Nice
France Hôpital Pitié-Salpêtrière Paris Ile-de-France
France Hôpitaux Universitaires de Strasbourg Strasbourg Grand Est
Germany University Hospital Bonn Clinic and Polyclinic for Neurology Bonn
Germany Medical Center of the University of Munich Friedrich Baur Institute at the Neurological Clinic and Polyclinic Munich
Hungary Semmelweis University Insitute of Genomics and Rare Disorders Budapest
Hungary University of Pécs Clinical Centre, Department of Neurology Pécs
Italy IRCCS Istituto delle Scienze Neurologiche Bologna
Italy A.O.U Policlinico di Messina U.O.C Neurologia e Malattie Neuromuscolari Messina Sicilia
Italy Fondazione IRCCS Istituto Neurologico "Carlo Besta" UOC Genetica Medica e Neurogenetica Milano Lombardia
Italy Azienda Ospedaliero-Universitaria Pisana Dipartimento di specialita' mediche UOC Neurologia Pisa Toscana
Italy Fondazione Policlinico Universitario Agostino Gemelli IRCCS Neurophysiopathology Unit Roma Lazio
Netherlands Radboud Universitair Medisch Centrum Nijmegen
New Zealand University of Auckland Auckland
Norway Haukeland University Hospital Bergen
Spain Hospital Clinic de Barcelona Barcelona
Spain Hospital Universitario 12 de Octubre Madrid
Spain Hospital Universitari i Politècnic La Fe Valencia
United Kingdom Queen Square Centre for Neuromuscular Diseases London Greater London
United Kingdom The Newcastle upon Tyne Hospitals NHS Foundation Trust Newcastle Upon Tyne Tyne And Wear
United Kingdom Salford Royal NHS Foundation Trust Salford Greater Manchester
United States Akron Children's Hospital Akron Ohio
United States Massachusetts General Hospital Boston Massachusetts
United States University of Texas SouthWestern Medical Center Dallas Texas
United States Myology Institute, University of Florida Gainesville Florida
United States Centre for the Treatment of Pediatric Neurodegenerative Disease, University of Texas McGovern Medical School Houston Texas
United States University of California, San Diego La Jolla California
United States Columbia University Irving Medical Center New York New York
United States The Children's Hospital of Philadelphia Philadelphia Pennsylvania
United States UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania

Sponsors (1)

Lead Sponsor Collaborator
Reneo Pharma Ltd

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Canada,  Czechia,  Denmark,  France,  Germany,  Hungary,  Italy,  Netherlands,  New Zealand,  Norway,  Spain,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change in Distance Walked During a 12 Minute Walk Test Distance walked in meters Baseline to Week 24
Secondary Change in PROMIS Short Form - Fatigue 13a (FACIT-fatigue) Scores The PROMIS is a 13-item questionnaire to describe fatigue and its impact upon daily activities and function. Each item is scored between 1=Not At All and 5=Very Much Baseline to Week 24
See also
  Status Clinical Trial Phase
Terminated NCT03323749 - A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension Phase 3
Terminated NCT04641962 - A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy Phase 2
Terminated NCT03862846 - A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy Phase 1
Terminated NCT05267574 - An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead) Phase 2/Phase 3