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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 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.

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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.

Conditions Studied

Methylmalonic Acidemia Propionic Acidemia, Type I and/or Type II Carbamoyl-Phosphate Synthase I Deficiency Disease Ornithine Carbamoyltransferase Deficiency

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

FieldValue
Enrollment Target 35 participants
Start Date 2012-09-01
Est. Completion 2020-04-30
Phase Phase 2

Sponsor

Mendel Tuchman

2 total trials

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What the Registry Record Tells You About NCT01599286

The ClinicalTrials.gov registry entry for NCT01599286 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 35 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 421-participant average among 6 other Methylmalonic Acidemia trials with a reported enrollment target (92% lower). The listed sponsor is Mendel Tuchman, which has 2 total studies on file at ClinicalTrials.gov.

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 reports 9 study locations spanning 7 distinct geographic areas - top geographies include 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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Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.