CompletedNot Applicable

Long-term Efficacy of Carglumic Acid in Organic Acidemia.

Taiwan3 participantsStarted 2019-11-28

Plain-language summary

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

Who can participate

Age range0 Years – 18 Years

SexALL

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Inclusion criteria

✓. PA or MMA confirmed using the following criteria:
✓. 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 criteria

✕. 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®

What they're measuring

Number of emergency visits due to hyperammonemia within 12 months period

Timeframe: 12 months

Trial details

NCT IDNCT04284917

SponsorNational Taiwan University Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-12-31

View on ClinicalTrials.gov More Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) trials