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 and Drug Administration (FDA) on 18 March 2010.
- 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
- Conditions
- Interventions
- 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
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
Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia
- First Posted Date
- 2015-04-27
- Last Posted Date
- 2019-04-25
- 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
- Conditions
- Interventions
- 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
Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
- First Posted Date
- 2011-04-25
- Last Posted Date
- 2014-05-29
- Lead Sponsor
- Children's Hospital of Philadelphia
- Registration Number
- NCT01341379
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
Drug Development Updates
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