Phase 1 N=4 Treatment

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

Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency

Bottom Line

View on ClinicalTrials.gov: NCT01881984 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Jul 2017

Primary outcome: Primary: Metabolic Stress

Study Design & Population

Study type: Interventional
Phase: Phase 1
Interventions: Ravicti (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: University of Pittsburgh
Primary completion: Feb 2016

Outcome Measures

Outcome	Result	p-value
PRIMARY Metabolic Stress	—	—
SECONDARY Pharmacokinetic (pK)Analysis	—	—

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. 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.

Eligibility Criteria

Inclusion Criteria

confirmation of a diagnosis of MCAD deficiency
at least one copy of 985A>G MCAD mutation
ability to follow protocol

Exclusion Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01881984). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.