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Phase 3 randomized double-blind placebo-controlled crossover study of triheptanoin for paroxysmal movement disorders in Glut1 deficiency syndrome
MOH_2017-06-05_000304Phase 3Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Jun 2017
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial of triheptanoin in Glucose Transporter Type 1 Deficiency Syndrome (Glut1DS) (2 arms), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Frequency of functionally impairing paroxysmal movement disorder events. Started Jun 2017; status: completed.
02
Drugs
triheptanoin
03
Conditions
Glucose Transporter Type 1 Deficiency Syndrome (Glut1DS)
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