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

17180

This is a prospective mixed-design study focused on the long-term management of propionic aciduria (PA) and methylmalonic aciduria (MMA) with N-carbamylglutamate (NCG) maintenance therapy. Treatment characteristics, clinical outcomes, and healthcare utilization data of patients diagnosed PA or MMA treated \>6 months therapy with NCG are collected at baseline, 12 months, 18 months, 36 months and 54 months. Qualitative interviews with adult patients and caregivers are conducted \>6 months after study enrollment to gain a better understanding of the disease burden and the treatment burden of patients and their families.

2019-01-15

2024-12-30

2029-07-30

Recruiting

95

Inclusion Criteria: 1. Patient has confirmed diagnosis of an organic acidemia (e.g., MMA or PA) 2. Patient initiated treatment with carglumic acid for long-term management of MMA or PA 3. Patient has been treated with carglumic acid for a minimum of 6 months 4. Patient (or caregiver) is able to comply with all prospective study procedures 5. Patient (or caregiver) is able to provide informed consent Exclusion Criteria: * None

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'CASE_ONLY', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 95, 'type': 'ESTIMATED'}}

2024-02-05