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NCT01599286 · ClinicalTrials.gov registry record · Phase 2
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
A Phase 2 study of Methylmalonic Acidemia and Propionic Acidemia, Type I and/or Type II, sponsored by Mendel Tuchman.
- Completed
- Registry status
- Phase 2
- Development phase
- 35
- Enrollment target
- 9
- Study locations
NCT01599286: Completed Phase 2 study of Methylmalonic Acidemia and Propionic Acidemia, Type I and/or Type II, sponsored by Mendel Tuchman.
NCT01599286 is a Phase 2 study of Methylmalonic Acidemia and Propionic Acidemia, Type I and/or Type II that has completed, run by Mendel Tuchman. The registered enrollment target is 35 participants, below the 421-participant average among 6 other Methylmalonic Acidemia trials with a reported enrollment target (92% lower). The trial reports 9 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01599286, a Phase 2 study of Methylmalonic Acidemia and Propionic Acidemia, Type I and/or Type II, has completed, sponsored by Mendel Tuchman.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 35 participants
- Enrollment target
- 9
- Study locations
Study Summary
The overall objective of this drug trial is to determine whether the treatment of acute hyperammonemia with N-carbamyl-L-glutamate (NCG, Carglumic acid) in propionic acidemia (PA), methylmalonic acidemia (MMA), late-onset CPS1 deficiency (CPSD) and late-onset Ornithine transcarbamylase deficiency (OTCD) accelerates the resolution of hyperammonemia efficiently and safely. The primary goal is to determine if the study drug (NCG) efficiently reduces ammonia levels following a hyperammonemia episode(s). Secondly, the investigators want to know if treatment with this study drug (NCG) efficiently improves neurologic function, reduces plasma glutamine levels and lessens the duration of hospitalization after each episode of hyperammonemia.
Primary Outcome
The composite primary intention to treat (ITT) outcome of the earlier of time to reach an ammonia level of ≤50 µmol/L or hospital discharge. Data presented as a hazard ratio based on the time to reach an ammonia level of ≤50 µmol/L. The outcome measure was a survival analysis based on time to reach the earlier of an ammonia level of ≤50 µmol/L or time to discharge, which was considered to be a point where the patient was no longer at risk of neurological injury from ammonia. The outcome of survi
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Carbaglu
Study Locations (9)
California
- University of California Los Angeles - Los Angeles
- Lucile Packard Children's Hospital at Stanford - Palo Alto
Pennsylvania
- The Children's Hospital of Philadelphia (CHOP) - Philadelphia
- University of Pittsburgh - Pittsburgh
Colorado
- The Children's Hospital of Colorado - Aurora
District of Columbia
- Children's National Medical Center - Washington D.C.
Massachusetts
- Children's Hospital Boston - Boston
New York
- Mount Sinai School of Medicine - New York
Ohio
- University Hospitals Cleveland Medical Center - Cleveland
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 35 participants |
| Start Date | 2012-09-01 |
| Est. Completion | 2020-04-30 |
| Phase | Phase 2 |
What the finished NCT01599286 record still lists
NCT01599286 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 35 participants, a relatively small participant target, below the 421-participant average among 6 other Methylmalonic Acidemia trials with a reported enrollment target (92% lower).
The record links to 4 conditions, with Methylmalonic Acidemia appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT01599286 lists 9 locations in 7 states (California, Pennsylvania, Colorado).
Frequently Asked Questions
What is clinical trial NCT01599286 about?
NCT01599286 is a clinical study titled "Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia". The overall objective of this drug trial is to determine whether the treatment of acute hyperammonemia with N-carbamyl-L-glutamate (NCG, Carglumic acid) in propionic acidemia (PA), methylmalonic acidemia (MMA), late-onset CPS1 deficiency (CPSD) and late-onset Ornithine transcarbamylase deficiency (O...
What is the current status of trial NCT01599286?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 35 participants. The study started on 2012-09-01. Estimated completion is 2020-04-30.
What conditions does trial NCT01599286 study?
This clinical trial studies the following conditions: Methylmalonic Acidemia, Propionic Acidemia, Type I and/or Type II, Carbamoyl-Phosphate Synthase I Deficiency Disease, Ornithine Carbamoyltransferase Deficiency.
What interventions are being tested in trial NCT01599286?
The interventions under investigation include: Placebo (DRUG), Carbaglu (DRUG).
Who is sponsoring clinical trial NCT01599286?
This trial is sponsored by Mendel Tuchman, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01599286 being conducted?
This trial has 9 study locations across California, Colorado, District of Columbia, Massachusetts, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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