NCT01881984View on ClinicalTrials.gov

Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation

PHASE1CompletedINTERVENTIONAL
Enrollment

4

Participants

Timeline

Start Date

June 30, 2013

Primary Completion Date

February 29, 2016

Study Completion Date

February 29, 2016

Conditions
Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
Interventions
DRUG

Ravicti

Open-label design comparing Ravicti at doses of 2, 4, and 6 grams/m2/day

Trial Locations (1)

15224

Children's Hospital of Pittsburgh of UPMC, Pittsburgh

All Listed Sponsors
collaborator

Horizon Pharma Ireland, Ltd., Dublin Ireland

INDUSTRY

lead

University of Pittsburgh

OTHER

NCT01881984 - Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation | Biotech Hunter | Biotech Hunter