Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation
Completed · Phase 1
Conditions studied: Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
In brief
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. The medication is glycerol phenylbutyrate, called Ravicti, which is currently FDA approved for the treatment of urea cycle disorders. Previous research suggests that Ravicti may also be effective in the treatment MCAD deficiency. This study will investigate the safety and efficacy (how well it works) of Ravicti in patients with MCAD deficiency caused by having at least one copy of the 985A\>G mutation.
Key facts
- Study ID
- NCT01881984
- Run by
- University of Pittsburgh
- People needed
- 4
- Starts
- 2013-06-01
- Expected to finish
- 2016-02-01
- Last updated by the study team
- 2017-09-25
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- confirmation of a diagnosis of MCAD deficiency
- at least one copy of 985A>G MCAD mutation
- ability to follow protocol
You may not qualify if…
- 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
Where it is running
- Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
Full record on ClinicalTrials.gov
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