A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy
- Registration Number
- NCT03862846
- Lead Sponsor
- Reneo Pharma Ltd
- Brief Summary
The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 23
Inclusion Criteria
- Subjects must give written, signed and dated informed consent
- Confirmed diagnosis of PMM according to the 2016 Rome Consensus recommendations
- Confirmed mitochondrial mutation with evidence of myopathy
- Able to remain on stable medication throughout the study
Exclusion Criteria
- Documented evidence of ongoing rhabdomyolysis
- Subjects with motor abnormalities other than related to mitochondrial disease
- Treatment with an investigational drug within 3 months prior to Day 1
- Hospitalised within 3 months prior to screening for any major medical condition
- Pregnant or nursing females
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Group REN001 REN001 Low Dose
- Primary Outcome Measures
Name Time Method Adverse Events Comparing Baseline to Week 12 Number of participants with Adverse Events as a measure of safety and tolerability
- Secondary Outcome Measures
Name Time Method Adverse Events Continous to Week 48 Number of participants with Adverse Events as a measure of safety and tolerability
Trial Locations
- Locations (2)
Wellcome Centre for Mitochondrial Research
🇬🇧Newcastle Upon Tyne, United Kingdom
Institute of Neurology, University College London
🇬🇧London, United Kingdom