An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

• Status: Recruiting
• Trial end date: 2025-07-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a long-term safety and tolerability study. REN001 is administered once daily to subjects with PMM who have previously completed Study REN001-201 or participated in Study REN001-101 and withdrew from that study due to the COVID-19 pandemic.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Reneo Pharma Ltd

Criteria

Inclusion Criteria:

• Completed treatment in STRIDE or was participating in Study REN001-101 when the study stopped due to the COVID-19 pandemic, and in the opinion of the Investigator and Sponsor had been compliant with the study requirements.

• Have PMM which continues to be primarily characterized by exercise intolerance or active muscle pain.

• Willing and able to swallow the REN001 gelatin capsules.

• 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.

• Signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.

• 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 the last dose of study drug. Males with partners who are women of childbearing potential (WOCBP) must also use contraception from baseline through to 14 weeks after the last dose of study drug.

Exclusion Criteria:

• Anticipated to need a peroxisome proliferator-activated receptor (PPAR) agonist other than REN001 during the study.

• Intent to donate blood, or blood components during the study or within one month after completion of the study.

• Current drug dependency. Use of opiates/cannabis for medical reasons is acceptable with prescription evidence or at the Investigator's discretion.

• Current alcohol dependency.

• Any medical, psychiatric or laboratory condition 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.

• Pregnant or nursing female

For subjects who enter after exiting Stride or REN001-101 studies the following exclusion criteria will also apply:

1. 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.

2. 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.

3. Subjects with uncontrolled diabetes and/or a Screening HbA1c of ≥11%.

4. 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.)

5. Subjects with a history of cancer. A history of in situ basal cell carcinoma in the skin is allowed.

6. Clinically significant cardiac disease and/or clinically significant ECG abnormalities such as 2nd degree heart block, symptomatic tachyarrhythmia or unstable arrhythmia (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.