An unblinded , multi-centre study to determine the long term safety and tolerability of the study drug REN001 in Primary Mitochondrial Myopathy (PMM) subjects.
- Conditions
- Primary Mitochondrial MyopathyMedDRA version: 20.0Level: PTClassification code 10027710Term: Mitochondrial myopathySystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2021-003471-34-ES
- Lead Sponsor
- Reneo Pharma Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 80
1. Completed treatment in the parent (STRIDE) study or was participating in Study REN001-101 when the study was stopped due to the COVID-19 pandemic, and in the opinion of the Investigator and the Sponsor have been compliant with the study requirements.
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 75
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5
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;Timepoint(s) of evaluation of this end point: throughout the study
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Absolute values, changes from baseline, and incidence of potentially clinically significant changes in:<br>•Laboratory safety tests<br>•Electrocardiograms (ECG)<br>•Supine vital signs<br>•Eye assessments<br><br>Absolute values and changes from baseline in:<br>•Distance walked during the 12-Minute Walk Test (12MWT)<br>•Modified Fatigue Impact Scale (MFIS) total scores and sub-scale scores<br>•Patient Global Impression of Severity (PGIS) scores for fatigue and muscle symptoms<br>•Brief Pain Inventory (BPI) pain severity and pain interference scores<br>•Patient Reported Outcomes Measurement Informal System (PROMIS) Short Form-Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue 13a scores<br>•36 Item Short Form Health Survey (SF-36) domain scores (7-day recall)<br>•Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP) scores<br>•PGIC (muscle and fatigue symptoms) scores;Timepoint(s) of evaluation of this end point: throughout the study