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EPI-743 in Children with Mitochondrial or Metabolic Disorders
NCT01642056Phase 2Completed
- Sponsor
- National Human Genome Research Institute
- Start
- Sep 2012
- Primary completion
- Sep 2019
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, randomised, double-blind trial of vatiquinone in mitochondrial disease and metabolic disorders (2 arms), sponsored by National Human Genome Research Institute. Primary endpoint: Newcastle Paediatric Mitochondrial Disease Scale Part I-III at 14 months. Started Sep 2012; status: completed.
02
Drugs
vatiquinone
03
Conditions
mitochondrial diseasemetabolic disorders
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