You're using a free limited version of DrugPatentWatch: Upgrade for Complete Access

Last Updated: November 4, 2024

CLINICAL TRIALS PROFILE FOR CARGLUMIC ACID


✉ Email this page to a colleague

« Back to Dashboard


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.
NCT01597440 ↗ Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia Terminated Children's 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.
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for CARGLUMIC ACID

Condition Name

Condition Name for CARGLUMIC ACID
Intervention Trials
Methylmalonic Acidemia 3
Propionic Acidemia 2
Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) 1
Propionic Acidemia, Type I and/or Type II 1
[disabled in preview] 0
This preview shows a limited data set
Subscribe for full access, or try a Trial

Condition MeSH

Condition MeSH for CARGLUMIC ACID
Intervention Trials
Propionic Acidemia 4
Amino Acid Metabolism, Inborn Errors 4
Acidosis 4
Carbamoyl-Phosphate Synthase I Deficiency Disease 1
[disabled in preview] 0
This preview shows a limited data set
Subscribe for full access, or try a Trial

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
This preview shows a limited data set
Subscribe for full access, or try a Trial

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
This preview shows a limited data set
Subscribe for full access, or try a Trial

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
[disabled in preview] 0
This preview shows a limited data set
Subscribe for full access, or try a Trial

Clinical Trial Status

Clinical Trial Status for CARGLUMIC ACID
Clinical Trial Phase Trials
Completed 2
Enrolling by invitation 1
Terminated 1
[disabled in preview] 0
This preview shows a limited data set
Subscribe for full access, or try a Trial

Clinical Trial Sponsors for CARGLUMIC ACID

Sponsor Name

Sponsor Name for CARGLUMIC ACID
Sponsor Trials
University Hospitals Cleveland Medical Center 2
University of California, Los Angeles 2
Mendel Tuchman 2
[disabled in preview] 4
This preview shows a limited data set
Subscribe for full access, or try a Trial

Sponsor Type

Sponsor Type for CARGLUMIC ACID
Sponsor Trials
Other 24
[disabled in preview] 0
This preview shows a limited data set
Subscribe for full access, or try a Trial

Make Better Decisions: Try a trial or see plans & pricing

Drugs may be covered by multiple patents or regulatory protections. All trademarks and applicant names are the property of their respective owners or licensors. Although great care is taken in the proper and correct provision of this service, thinkBiotech LLC does not accept any responsibility for possible consequences of errors or omissions in the provided data. The data presented herein is for information purposes only. There is no warranty that the data contained herein is error free. thinkBiotech performs no independent verification of facts as provided by public sources nor are attempts made to provide legal or investing advice. Any reliance on data provided herein is done solely at the discretion of the user. Users of this service are advised to seek professional advice and independent confirmation before considering acting on any of the provided information. thinkBiotech LLC reserves the right to amend, extend or withdraw any part or all of the offered service without notice.