Primary Mitochondrial Myopathy
Conditions
Brief summary
The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy
Interventions
DRUGREN001
Once daily
Sponsors
Reneo Pharma Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
16 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse Events | Comparing Baseline to Week 12 | Number of participants with Adverse Events as a measure of safety and tolerability |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Adverse Events | Continous to Week 48 | Number of participants with Adverse Events as a measure of safety and tolerability |
Countries
United Kingdom
Outcome results
None listed