An unblinded , multi-centre study to determine the long term safety and tolerability of the study drug REN001 in Primary Mitochondrial Myopathy (PMM) subjects.

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 200

Inclusion Criteria

1. mtDNA-PMM subjects: Completed treatment in STRIDE or participated in Study REN001-101, and in the opinion of the Investigator and Sponsor had been compliant with the study requirements.
or
nDNA-PMM subjects: Subjects aged 18 years or older with known nuclear (nDNA) pathogenic variants with a major muscle phenotype consisting of objective myopathy with poor exercise tolerance. Proof of
pathogenicity must be provided. Must be able to walk at least 100m in the screening 12MWT and the limitations in walk test must be primarily due to the energy deficit and not due to ataxia or any other condition. For subjects under 25 years old only: confirmation of bone growth plate
closure by wrist radiograph.
2. Have PMM which continues to be primarily characterized by exercise intolerance or active muscle pain.
3. Willing and able to swallow gelatin capsules.
4. Concomitant medications (including supplements) intended for the treatment of PMM or other co-morbidities likely to remain stable throughout participation in the study where clinically possible.
5. Signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
6. Females should be either of non-child-bearing potential or must agree to use highly effective methods of contraception from baseline through to approximately 30 days after last dose of study drug. Males with partners who are WOCBP must also use contraception from baseline through to 14 weeks after last dose of study drug.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 180
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion Criteria

1. Anticipated to need a PPAR agonist other than REN001 during the study.
2. Anticipated to need drugs during the study with a narrow therapeutic index and Breast Cancer Resistant Protein (BCRP) mediated absorption, distribution, metabolism and excretion (ADME).
3. Intent to donate blood, or blood components during the study or within one month after completion of the study.
4. Current drug dependency. Use of opiates/cannabis for medical reasons is acceptable with prescription evidence or at the Investigator’s discretion.
5. Current alcohol dependency.
6. Any medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or interfere with the interpretation of study results and, in the judgment of the Investigator and Medical Monitor, would make the subject inappropriate for entry into this study.
7. Pregnant or nursing females.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate long-term safety and tolerability of REN001 in subjects with PMM.;Secondary Objective: To assess patients with PMM who are receiving long-term treatment with REN001 in terms of PMM associated symptoms, exercise endurance, quality of life (QoL) and work productivity.;Primary end point(s): Safety and tolerability of REN001 as assessed by:<br>•Number and severity of adverse events (AE)<br>•Number of AEs leading to study drug discontinuation<br>•Number of serious adverse events (SAEs) <br>•Number of adverse events of special interest (AESIs)<br>•Number of AEs leading to death<br>;Timepoint(s) of evaluation of this end point: throughout the study

Secondary Outcome Measures

Name	Time	Method

An unblinded , multi-centre study to determine the long term safety and tolerability of the study drug REN001 in Primary Mitochondrial Myopathy (PMM) subjects.

Recruitment & Eligibility

Study & Design

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AN OPEN-LABEL, MULTI-CENTRE STUDY TO EVALUATE THE LONG-TERM SAFETY AND TOLERABILITY OF REN001 IN SUBJECTS WITH PRIMARY MITOCHONDRIAL MYOPATHY (PMM)