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

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.

NCT Number	NCT04176523
Lead Sponsor	RECORDATI GROUP
Conditions	Methylmalonic Acidemia, Propionic Acidemia
Enrollment	95 participants
Start Date	2019-01-15
Primary Completion	2024-12-30 (estimated)
Study Completion	2029-07-30 (estimated)
Updated on ClinicalTrials.gov	2024-02-05