An interventional study of Carglumic Acid (Carbaglu®) in Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA), sponsored by National Taiwan University Hospital. Completed at 1 site in Taiwan. Open to participants aged 0 Years to 18 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2025-03-20.
Sponsored by National Taiwan University Hospital · Not applicable, Interventional, and Treatment
Evaluate the Long Term Effectiveness \& Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).
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, 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.
23 studies on the registry are indexed under Propionic Acidemia; 8 are open to participants now.
This study's enrollment of 3 is below the median of 30 across 12 interventional studies indexed under Propionic Acidemia.
Browse Propionic Acidemia studies →National Taiwan University Hospital is the lead sponsor of 2,615 studies on the registry; 576 are open to participants now.
Of its 11 completed or terminated interventional studies of FDA-regulated products, 2 (18%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
PA or MMA confirmed using the following criteria:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Experimental Case_ Carglumic Acid
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.
Number of emergency visits due to hyperammonemia within 12 months period
Adverse Events
Time frame: 12 months
Time to first visit to the ER due to hyperammonemia from starting the treatment
Adverse Events
Time frame: 12 months
Assessment of Plasma ammonia level
Plasma ammonia level over the study treatment period.
Time frame: Baseline,3,6,9 and 12,months after receiving Carbaglu.
Number of days of hospitalization
Number of days of hospitalization during study treatment period.
Time frame: 12 months
Assessment of Acylcarnitine level
Acylcarnitine level for all patients
Time frame: Baseline,3,6,9 and 12,months after receiving Carbaglu.
Assessment of urine organic acid level
Measuring urine organic acid level for both diseases.
Time frame: Baseline,3,6,9 and 12,months after receiving Carbaglu.
Assessment of Plasma aminoacids' level
Measuring Plasma aminoacids' level for both diseases.
Time frame: Baseline,3,6,9 and 12,months after receiving Carbaglu.
Assessment of Methylmalonic acid level
Measuring Methylmalonic acid level for both diseases.
Time frame: Baseline,3,6,9 and 12,months after receiving Carbaglu.
This study is completed, as verified in Mar 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Carglumic acid
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