CI

At a glance

ClinicalIndex Comparison Record
Phase 1Completed· 4 enrolled
Drug / intervention
Ravictidrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT01881984
NCT01881984Phase 1Completed
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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

University of Pittsburgh·interventional·Posted Jun 20, 2013·Updated Sep 25, 2017

In Brief

A Phase 1 clinical trial evaluating Ravicti for Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency. Completed, enrolled 4 participants across 1 site.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMedium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
CountriesUnited States
CollaboratorsHorizon Pharma Ireland, Ltd., Dublin Ireland

Timeline

Phase 1CompletedFinished
2014201520162017201820192020202120222023202420252026
First PostedJun 20, 2013
Enrollment StartJun 1, 2013
Primary CompletionFeb 1, 2016
TodayJul 2, 2026
Enrollment to primary: 2.7 yearsPosted 13.0 years ago

Interventions

Ravictidrug

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