An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Recruiting now
Conditions studied: Hyperammonemia, Methylmalonic Acidemia, Propionic Acidemia
In brief
To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
Key facts
- Study ID
- NCT05040178
- Run by
- RECORDATI GROUP
- People needed
- 20
- Starts
- 2022-06-30
- Expected to finish
- 2032-06-30
- Last updated by the study team
- 2025-04-15
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Provision of signed and dated informed consent/assent form
- Prescribed and treated with Carbaglu®
- Have an established diagnosis of PA or MMA defined as follows:
- Diagnosed with PA by semi quantitative urine organic acid analysis, defined as presence of elevated methylcitric acid and normal methylmalonic acid levels and no evidence of biotin related disorders in the organic acid analysis; OR
- Diagnosed with MMA by semi quantitative urine organic acid analysis, defined as elevation of methylmalonic acid and no evidence of vitamin B12 dependent disorder on plasma amino acid analysis (vitamin B12 dependency is defined by documented vitamin B12 responsiveness).
- AND/OR
- Confirmation by molecular genetic testing
You may not qualify if…
- None
Where it is running
- Children's National Hospital — Washington D.C., District of Columbia, United States (enrolling)
- University of South Florida — Tampa, Florida, United States (enrolling)
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
- Riley Children's Hospital — Indianapolis, Indiana, United States (enrolling)
- Icahn School of Medicine at Mt. Sinai — New York, New York, United States (enrolling)
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.