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Long-term Efficacy of Carglumic Acid in Organic Acidemia.

NCT04284917 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2025-03-20

No results posted yet for this study

Summary

Evaluate the Long Term Effectiveness \& Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).

Conditions

  • Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA)

Interventions

DRUG

Carglumic Acid (Carbaglu®)

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, 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 and methylmalonic acid will be recorded during treatment period.

Sponsors & Collaborators

  • National Taiwan University Hospital

    lead OTHER

Principal Investigators

  • Ni-Chung Lee, M.D., Ph.D · National Taiwan University Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2019-11-28
Primary Completion
2024-12-31
Completion
2025-03-11

Countries

  • Taiwan

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04284917 on ClinicalTrials.gov