A Phase 3 interventional study of Carglumic Acid in Propionic Acidemia and Methylmalonic Acidemia, sponsored by King Abdullah International Medical Research Center. Completed at 2 sites in Saudi Arabia. Open to participants aged Up to 15 Years. Per ClinicalTrials.gov, last updated 2019-04-25.
Sponsored by King Abdullah International Medical Research Center · Phase 3, Interventional, and Treatment
A Phase IIIb (Three b), Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness \& Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA). Carbaglu® clinical experience in Organic Acidemia (OA) is limited to a non-comparative retrospective collection of data from patients who had received Carbaglu® for 1 to 15 days.
There is no current evidence supporting the use of carglumic acid for the chronic management of patients with OA. The investigators are proposing a randomized multicentre prospective clinical trial to evaluate long-term effects of the use of Carbaglu® (50mg/kg/day) combined with standard chronic therapy in patients with PA and MMA compared to standard chronic therapy alone.
23 studies on the registry are indexed under Propionic Acidemia; 8 are open to participants now.
This study's enrollment of 33 is close to the median of 30 across 12 interventional studies indexed under Propionic Acidemia.
Browse Propionic Acidemia studies →King Abdullah International Medical Research Center is the lead sponsor of 46 studies on the registry; 6 are open to participants now.
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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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
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.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
patients with Methylmalonic Acidemia will receive standard therapy only (protein restricted diet, L-carnitine, metronidazole and vitamin B12)
patients with Methylmalonic Acidemia will receive Carglumic Acid in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and vitamin B12)
Drug: Carglumic Acid
patients with Propionic Acidemia will receive standard therapy only (protein restricted diet, L-carnitine, metronidazole and biotin)
patients with Propionic Acidemia will receive Carglumic Acid in addition to standard therapy (protein restricted diet, L-carnitine, metronidazole and biotin)
Drug: Carglumic Acid
Carglumic Acid 50 mg/kg/day (twice daily)
Also known as: Carbaglu
Number of emergency visits due to hyperammonemia within 24 months period
Time frame: 24 months
Time to first visit to the ER due to hyperammonemia from starting the treatment.
Time frame: 24 months
Plasma ammonia levels over the study treatment period.
Time frame: 24 months
Number of days of hospitalization
Time frame: 24 months
Acylcarnitine level for all patients
Time frame: once on screening visit
Measuring urine organic acid levels for both diseases.
Time frame: 24 months
Measuring Plasma aminoacids' levels for both diseases
Time frame: 24 months
This study is completed, as verified in Apr 2019. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Carglumic acid
King Abdullah International Medical Research Center