Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT01881984 · ClinicalTrials.gov registry record · Phase 1
Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation
A Phase 1 study, sponsored by University of Pittsburgh.
- Completed
- Registry status
- Phase 1
- Development phase
- 4
- Enrollment target
NCT01881984: Completed Phase 1 study, sponsored by University of Pittsburgh.
NCT01881984 is a Phase 1 study that has completed, run by University of Pittsburgh. The registered enrollment target is 4 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (93% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01881984, a Phase 1 study, has completed, sponsored by University of Pittsburgh.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 4 participants
- Enrollment target
Study 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.
Primary Outcome
Changes in the assessments of metabolic stress pre- and post-dosing with Ravicti will be the main outcome variable.
Interventions
- DRUG Ravicti
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2013-06 |
| Est. Completion | 2016-02 |
| Phase | Phase 1 |
What the finished NCT01881984 record still lists
NCT01881984 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 4 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (93% lower).
The record links to 0 conditions, and to 1 intervention - of which Ravicti is the first listed.
NCT01881984 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01881984 about?
NCT01881984 is a clinical study titled "Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation". 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 ...
What is the current status of trial NCT01881984?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2013-06. Estimated completion is 2016-02.
What interventions are being tested in trial NCT01881984?
The interventions under investigation include: Ravicti (DRUG).
Who is sponsoring clinical trial NCT01881984?
This trial is sponsored by University of Pittsburgh, which has 1,238 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01881984's enrollment target sits among peer trials
4 1995th of 2000 higher than 2 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia