Clinical trial
A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations
Name
CARBAGLU-RRDUS-PASS-0573
Description
To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
Trial arms
Trial start
2022-06-30
Estimated PCD
2032-06-30
Trial end
2032-06-30
Status
Recruiting
Treatment
Carglumic Acid
Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.
Arms:
Male and Female Adult and Pediatric Participants
Other names:
Carbaglu®
Size
20
Primary endpoint
Effects of Carbaglu® on plasma ammonia levels
Patients treated with Carbaglu® will be managed chronically (out-patient) or acutely (hospital in-patient). In both cases, data will be collected for approximately 1 year following discontinuation of Carbaglu treatment.
Adverse Event frequency and severity
Patients treated with Carbaglu® will be managed chronically (out-patient) or acutely (hospital in-patient). In both cases, data will be collected for approximately 1 year following discontinuation of Carbaglu treatment.
Eligibility criteria
Inclusion Criteria:
1. Provision of signed and dated informed consent/assent form
2. Prescribed and treated with Carbaglu®
3. 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
Protocol
{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'OTHER'}, 'enrollmentInfo': {'count': 20, 'type': 'ESTIMATED'}}
Updated at
2024-01-30
1 organization
1 product
3 indications
Product
Carglumic AcidIndication
HyperammonemiaIndication
Methylmalonic acidemiaIndication
Propionic AcidemiaOrganization
Recordati Rare Diseases