Status:
COMPLETED
Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation
Lead Sponsor:
University of Pittsburgh
Collaborating Sponsors:
Horizon Pharma Ireland, Ltd., Dublin Ireland
Conditions:
Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief Summary
This is a medical research study to test a medication in adult patients with a disease called medium-chain acyl-CoA dehydrogenase (MCAD) deficiency caused by at least one copy of the 985A\>G mutation....
Detailed Description
Participation in the study will require one overnight admission and three outpatient visits at the Clinical and Translational Research Center at Children's Hospital of Pittsburgh of UPMC (also called ...
Eligibility Criteria
Inclusion
- confirmation of a diagnosis of MCAD deficiency
- at least one copy of 985A\>G MCAD mutation
- ability to follow protocol
Exclusion
- positive pregnancy test
- currently breastfeeding
- currently taking any medication for which there is a potential drug interaction with Ravicti, includes corticosteroids, valproic acid, haloperidol, and probenecid
- liver or kidney insufficiency
Key Trial Info
Start Date :
June 1 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2016
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT01881984
Start Date
June 1 2013
End Date
February 1 2016
Last Update
September 25 2017
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224