Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
RECORDATI GROUP
95 participants
Jan 15, 2019
OBSERVATIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria5
- Patient has confirmed diagnosis of an organic acidemia (e.g., MMA or PA)
- Patient initiated treatment with carglumic acid for long-term management of MMA or PA
- Patient has been treated with carglumic acid for a minimum of 6 months
- Patient (or caregiver) is able to comply with all prospective study procedures
- Patient (or caregiver) is able to provide informed consent
Exclusion Criteria1
- None
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Interventions
Maintenance therapy with carglumic acid
Locations(32)
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NCT04176523