Carglumic acid

Generic Name
Carglumic acid
Brand Names
Carbaglu, Ucedane
Drug Type
Small Molecule
Chemical Formula
C6H10N2O5
CAS Number
1188-38-1
Unique Ingredient Identifier
5L0HB4V1EW
Background

Carglumic acid is a drug used for the treatment of hyperammonemia in patients with a deficiency in N-acetyl glutamate synthase. This rare genetic disorder results in elevated blood levels of ammonia, which can eventually cross the blood–brain barrier and cause neurologic problems, cerebral edema, coma, and death. Carglumic acid was approved by the U.S. Food ...

Indication

For the treatment of acute and chronic hyperammonaemia in patients with N-acetylglutamate synthase (NAGS) deficiency. This enzyme is an important component of the urea cycle to prevent build up of neurotoxic ammonium in the blood.

Associated Conditions
Acute Hyperammonaemia, Chronic Hyperammonaemia
Associated Therapies
Maintenance therapy

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

First Posted Date
2021-09-10
Last Posted Date
2024-01-30
Lead Sponsor
Recordati Rare Diseases
Target Recruit Count
20
Registration Number
NCT05040178
Locations
🇺🇸

Children's National Hospital, Washington, District of Columbia, United States

🇺🇸

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States

🇺🇸

University of South Florida, Tampa, Florida, United States

and more 2 locations

Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach

First Posted Date
2019-11-25
Last Posted Date
2024-02-05
Lead Sponsor
Recordati Rare Diseases
Target Recruit Count
95
Registration Number
NCT04176523
Locations
🇫🇷

Hopital de Hautepierre, Strasbourg, France

🇮🇹

Azienda Ospedaliero Universitaria Meyer, Firenze, Italy

🇩🇪

Klinikum Bremen Mitte, Prof. Hess Kinderklinik, Bremen, Germany

and more 29 locations

Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

First Posted Date
2015-04-27
Last Posted Date
2019-04-25
Lead Sponsor
King Abdullah International Medical Research Center
Target Recruit Count
33
Registration Number
NCT02426775
Locations
🇸🇦

King Fahad Medical City, Riyadh, Saudi Arabia

🇸🇦

King Abdullah Specialist Children Hospital, King Abdulaziz Medical City, Riyadh, Saudi Arabia

Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia

First Posted Date
2012-05-14
Last Posted Date
2017-04-26
Lead Sponsor
Mendel Tuchman
Target Recruit Count
1
Registration Number
NCT01597440
Locations
🇺🇸

University of California Los Angeles, Los Angeles, California, United States

🇺🇸

Lucile Packard Children's Hospital at Stanford, Palo Alto, California, United States

🇺🇸

The Children's Hospital of Colorado, Aurora, Colorado, United States

and more 4 locations

N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia

First Posted Date
2009-02-13
Last Posted Date
2020-02-17
Lead Sponsor
Mendel Tuchman
Target Recruit Count
52
Registration Number
NCT00843921
Locations
🇺🇸

Childrens Research Institute, Washington, District of Columbia, United States

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