RG2133 (2',3',5'-Tri-O-Acetyluridine) in Mitochondrial Disease
Phase 1
Terminated
- Conditions
- Mitochondrial Diseases
- Registration Number
- NCT00060515
- Lead Sponsor
- Repligen Corporation
- Brief Summary
The objective of the study is to determine the safety and tolerability of RG2133 in patients with Mitochondrial Disease.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 12
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
What molecular mechanisms does 2',3',5'-Tri-O-Acetyluridine target in inherited mitochondrial diseases?
How does RG2133 compare to standard-of-care treatments for mitochondrial myopathy and encephalopathy?
Which biomarkers correlate with safety and efficacy outcomes in NCT00060515 mitochondrial disease trials?
What adverse events are associated with nucleoside analog therapy in mitochondrial DNA maintenance disorders?
Are there combination therapies involving RG2133 and ETC modulators for mitochondrial complex deficiencies?