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Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
NCT01599286 · View on ClinicalTrials.gov ↗
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01599286
The ClinicalTrials.gov registry entry for NCT01599286 describes a study currently listed as completed. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 35 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Mendel Tuchman, which has 1 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT01599286 reports 9 study locations spanning 7 distinct geographic areas — top geographies include California, Pennsylvania, Colorado. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT01599286?
The interventions under investigation include: Placebo (DRUG), Carbaglu (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT01599286?
This trial is sponsored by Mendel Tuchman, which has 1 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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