Status:
COMPLETED
Long-term Efficacy of Carglumic Acid in Organic Acidemia.
Lead Sponsor:
National Taiwan University Hospital
Conditions:
Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)
Eligibility:
All Genders
Up to 18 years
Phase:
NA
Brief Summary
Evaluate the Long Term Effectiveness \& Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).
Detailed Description
Study procedures: A. Study period: 36 months; treatment period: 12 months. B. Patients will receive Carglumic Acid (Carbaglu®) 50 mg/kg/day in addition to standard therapy (protein restricted diet, ...
Eligibility Criteria
Inclusion
- PA or MMA confirmed using the following criteria:
- PA confirmed by the measurement of acylcarnitine profile, urine organic acid, measurement of propionyl Co-A carboxylase in leukocytes or cultured fibroblasts or by DNA molecular testing of PCCA (Propionyl CoA Carboxylase, Alpha Polypeptide) or PCCB (propionyl CoA carboxylase, beta polypeptide) gene
- MMA confirmed by the measurement of acylcarnitine profile, urine organic acid, measurement of methymalonyl Co-A mutase in culture fibroblasts or DNA molecular testing of mutgene.
- Male or female Children of 18 years old or less.
- Had experienced Hyperammonemia(NH3 ≧100 uM) before.
- Not participating in any other clinical trial in the previous 30 days
Exclusion
- Patients with other organic acidemia or any other cause of hyperammonemia
- Patient receiving other investigational therapy for PA or MMA
- Patient with PA or MMA and other inherited genetic conditions or congenital anomalies
- Past history of hypersensitivity or drug allergy to Carbaglu®
Key Trial Info
Start Date :
November 28 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 11 2025
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT04284917
Start Date
November 28 2019
End Date
March 11 2025
Last Update
March 20 2025
Active Locations (1)
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1
National Taiwan University Hospital
Taipei, Taiwan, 10041