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Evaluation of Triheptanoin on fatty acid oxidation and exercise tolerance in glycogen storage diseases
2017-004153-17Phase 2Terminated
- Sponsor
- Neuromuscular Research Unit
- Start
- Mar 2018
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, randomised, double-blind trial of triheptanoin in Glycogen storage disease type III, Glycogenin-1 Related Myopathy and glycogen storage disease type VII (GSDVII) (2 arms), sponsored by Neuromuscular Research Unit. Primary endpoint: Heart rate during constant load cycling exercise (HR_const). Started Mar 2018; status: terminated.
02
Drugs
triheptanoin
03
Conditions
Glycogen storage disease type IIIGlycogenin-1 Related Myopathyglycogen storage disease type VII (GSDVII)
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