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

NCT01881984 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2017-09-25

Study results available
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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.

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

Sponsors & Collaborators

  • Horizon Pharma Ireland, Ltd., Dublin Ireland

    collaborator INDUSTRY

  • University of Pittsburgh

    lead OTHER

Principal Investigators

  • Gerard Vockley, MD, PhD · University of Pittsburgh/Children's Hospital of Pittsburgh of UPMC

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-06-30
Primary Completion
2016-02-29
Completion
2016-02-29

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01881984 on ClinicalTrials.gov