Primary Mitochondrial Myopathy
5
0
0
1
Key Insights
Highlights
Treatment Research
5 treatments under study, led by Ren001
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 65/100
80.0%
4 terminated out of 5 trials
20.0%
-66.6% vs benchmark
20%
1 trials in Phase 3/4
400%
4 of 1 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 1 completed trials
Clinical Trials (5)
A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy
An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)
An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients
A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension
A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy
Research Network
Common Treatments
- Ren0012
- Mavodelpar1
- Elamipretide Open Label Treatment1
- Bocidelpar1
- Elamipretide1
Active Sponsors
- Reneo Pharma Ltd3
- Astellas Pharma Inc1
- Stealth BioTherapeutics Inc.1
Top Investigators
Massachusetts General Hospital (MGH)
Newcastle Hospital NHS Foundation Trust
Wellcome Centre for Mitochondrial Research