CLINICAL TRIALS PROFILE FOR CARGLUMIC ACID

All Clinical Trials for CARGLUMIC ACID

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01597440 ↗	Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia	Terminated	Boston Children's Hospital	Phase 2	2012-09-01	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.
NCT01597440 ↗	Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia	Terminated	Boston Children’s Hospital	Phase 2	2012-09-01	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.
NCT01597440 ↗	Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia	Terminated	Children's Hospital of Philadelphia	Phase 2	2012-09-01	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.
NCT01597440 ↗	Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia	Terminated	Children's National Research Institute	Phase 2	2012-09-01	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.

Clinical Trial Conditions for CARGLUMIC ACID

Condition Name

Condition Name for CARGLUMIC ACID
Intervention	Trials
Methylmalonic Acidemia	3
Propionic Acidemia	2
Propionic Acidemia (PA)	1
Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)	1

Condition MeSH

Condition MeSH for CARGLUMIC ACID
Intervention	Trials
Propionic Acidemia	4
Amino Acid Metabolism, Inborn Errors	4
Acidosis	4
Ornithine Carbamoyltransferase Deficiency Disease	1

Clinical Trial Locations for CARGLUMIC ACID

Trials by Country

Trials by Country for CARGLUMIC ACID
Location	Trials
United States	13
Saudi Arabia	1
Taiwan	1

Trials by US State

Trials by US State for CARGLUMIC ACID
Location	Trials
Pennsylvania	2
Ohio	2
Massachusetts	2
District of Columbia	2
Colorado	2

Clinical Trial Progress for CARGLUMIC ACID

Clinical Trial Phase

Clinical Trial Phase for CARGLUMIC ACID
Clinical Trial Phase	Trials
Phase 3	1
Phase 2	2
N/A	1

Clinical Trial Status

Clinical Trial Status for CARGLUMIC ACID
Clinical Trial Phase	Trials
Completed	2
Enrolling by invitation	1
Terminated	1

Clinical Trial Sponsors for CARGLUMIC ACID

Sponsor Name

Sponsor Name for CARGLUMIC ACID
Sponsor	Trials
University of California, Los Angeles	2
Mendel Tuchman	2
Boston Children's Hospital	2

Sponsor Type

Sponsor Type for CARGLUMIC ACID
Sponsor	Trials
Other	24