Primary Mitochondrial Myopathy Clinical Trial
Official title:
An Open-label Study to Evaluate the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Patients With Primary Mitochondrial Myopathy (PMM), With an Optional Extension of Treatment
Verified date | March 2020 |
Source | Reneo Pharma Ltd |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy
Status | Terminated |
Enrollment | 23 |
Est. completion date | April 23, 2020 |
Est. primary completion date | April 23, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 16 Years and older |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
United Kingdom | Institute of Neurology, University College London | London | |
United Kingdom | Wellcome Centre for Mitochondrial Research | Newcastle Upon Tyne |
Lead Sponsor | Collaborator |
---|---|
Reneo Pharma Ltd |
United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Adverse Events | Number of participants with Adverse Events as a measure of safety and tolerability | Comparing Baseline to Week 12 | |
Secondary | Adverse Events | Number of participants with Adverse Events as a measure of safety and tolerability | Continous to Week 48 |
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