Long-term Efficacy of Carglumic Acid in Organic Acidemia.
Last updated on July 2021Recruitment
- Recruitment Status
- Enrolling by invitation
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)
- Type
- Interventional
- Phase
- Not Applicable
- Design
- Allocation: N/AIntervention Model: Single Group AssignmentMasking: None (Open Label)Primary Purpose: Treatment
Participation Requirements
- Age
- Between 18 years and 125 years
- Gender
- Both males and females
Description
Study procedures: A. Study period: 24 months; treatment period: 12 months. B. Patients will receive Carglumic Acid (Carbaglu®) 50 mg/kg/day in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and vitamin B12). C. Patients need to regularly come back for follow up exa...
Study procedures: A. Study period: 24 months; treatment period: 12 months. B. Patients will receive Carglumic Acid (Carbaglu®) 50 mg/kg/day in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and vitamin B12). C. Patients need to regularly come back for follow up examination on Day 0, month 3, month 6, and month 12. D. Patients should report any adverse event that occur during treatment period. E. Patients' number of emergency visits due to hyperammonemia, ammonia level, acylcarnitine, urinary organic acid analysis, plasma amino acid analysis will be recorded during treatment period.
Tracking Information
- NCT #
- NCT04284917
- Collaborators
- Not Provided
- Investigators
- Principal Investigator: Ni-Chung Lee, M.D., Ph.D National Taiwan University Hospital
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