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NCT01597440 · ClinicalTrials.gov registry record · Phase 2
Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia
A Phase 2 study, sponsored by Mendel Tuchman.
- Terminated
- Registry status
- Phase 2
- Development phase
- 1
- Enrollment target
NCT01597440 is a Phase 2 study that was terminated before completion, run by Mendel Tuchman. The registered enrollment target is 1 participants.
The verdict
NCT01597440, a Phase 2 study, was terminated before completion, sponsored by Mendel Tuchman.
- TERMINATED
- Registry status
- Phase 2
- Development phase
- 1 participants
- Enrollment target
Study Summary
Background: Very few drugs exist that treat hyperammonemia, specifically PA and MMA. Diet restrictions and alternate pathway agents are the current primary treatments, but they frequently fail to prohibit brain damage. Orthotopic liver transplantation cures the hyperammonemia of urea cycle disorders, but organ availability is limited and the procedure is highly invasive and requires life-long immunosuppression. A drug that could repair or stimulate a dysfunctional urea cycle such as this would have several advantages over current therapy. A drug called N-carbamyl-L-glutamate, Carglumic acid (NCG or Carbaglu)has recently been found to be virtually curative of another urea cycle defect called NAGS deficiency. In this disorder, treatment with NCG alone normalizes ureagenesis, blood ammonia and glutamine levels, allows normal protein tolerance and restores health. Knowledge from this study is being applied to acquired hyperammonemia, specifically in patients with propionic PA and MMA, to try and improve neurodevelopmental outcomes by improving the hyperammonemia. Aims: The overall objective of this project is to determine whether treatment of acute hyperammonemia with Carglumic acid in propionic acidemia (PA), methylmalonic acidemia (MMA) changes the long-term outcome of disease and to determine if it is effective in restoring urine ammonia levels to normal levels.
Interventions
- OTHER Standard of Care
- DRUG N-carbamylglutamate
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2012-09 |
| Est. Completion | 2015-02 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01597440
The ClinicalTrials.gov registry entry for NCT01597440 describes a study currently listed as terminated, categorized as Phase 2. The registered enrollment target is 1 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Mendel Tuchman, which has 2 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which Standard of Care is the first listed.
NCT01597440 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT01597440 about?
NCT01597440 is a clinical study titled "Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia". Background: Very few drugs exist that treat hyperammonemia, specifically PA and MMA. Diet restrictions and alternate pathway agents are the current primary treatments, but they frequently fail to prohibit brain damage. Orthotopic liver transplantation cures the hyperammonemia of urea cycle disorder...
What is the current status of trial NCT01597440?
This trial is currently terminated. It is a Phase 2 study. The enrollment target is 1 participants. The study started on 2012-09. Estimated completion is 2015-02.
What interventions are being tested in trial NCT01597440?
The interventions under investigation include: Standard of Care (OTHER), N-carbamylglutamate (DRUG).
Who is sponsoring clinical trial NCT01597440?
This trial is sponsored by Mendel Tuchman, which has 2 total clinical trials registered on ClinicalTrials.gov.
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