Clinical trial
Use of Glycerol Phenylbutyrate (Ravicti™) as a Chaperone to Stabilize Enzyme in Patients With MCAD Deficiency Due to the Common MCAD 985A>G (K304E) Mutation
NCT01881984 is registered in ClinicalTrials.gov with University of Pittsburgh as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- Use of Glycerol Phenylbutyrate (Ravicti™) as a Chaperone to Stabilize Enzyme in Patients With MCAD Deficiency Due to the Common MCAD 985A>G (K304E) Mutation
- Lead sponsor
- University of Pittsburgh
- Phase
- Phase 1
- Overall status
- Completed
- Start date
- June 30, 2013
- Completion date
- February 29, 2016
- Enrollment
- 4 (actual)
- Sites
- 1
Conditions
As the registry lists them; a condition with its own page is linked.
medium-chain acyl-coa dehydrogenase (mcad) deficiency