Status:
RECRUITING
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Lead Sponsor:
RECORDATI GROUP
Collaborating Sponsors:
Target PharmaSolutions, Inc.
Conditions:
Hyperammonemia
Methylmalonic Acidemia
Eligibility:
All Genders
Brief Summary
To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
Detailed Description
This study is being conducted to obtain short-term and long-term clinical safety information from adult and pediatric patients treated for hyperammonemia due to Methylmalonic Acidemia (MMA) and Propio...
Eligibility Criteria
Inclusion Criteria:
-
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
Exclusion Criteria:
- None
Key Trial Info
Start Date :
June 30 2022
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
June 30 2032
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT05040178
Start Date
June 30 2022
End Date
June 30 2032
Last Update
April 15 2025
Active Locations (5)
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1
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
2
University of South Florida
Tampa, Florida, United States, 33606
3
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
4
Riley Children's Hospital
Indianapolis, Indiana, United States, 46202