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An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

Recruitingn/aAges any - anyAllRECORDATI GROUP
NCT05040178 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.

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📋 Eligibility rules (as published)
Inclusion Criteria: 1. Provision of signed and dated informed consent/assent form 2. Prescribed and treated with Carbaglu® 3. Have an established diagnosis of PA or MMA defined as follows: * Diagnosed with PA by semi quantitative urine organic acid analysis, defined as presence of elevated methylcitric acid and normal methylmalonic acid levels and no evidence of biotin related disorders in the organic acid analysis; OR * Diagnosed with MMA by semi quantitative urine organic acid analysis, defined as elevation of methylmalonic acid and no evidence of vitamin B12 dependent disorder on plasma amino acid analysis (vitamin B12 dependency is defined by documented vitamin B12 responsiveness). AND/OR * Confirmation by molecular genetic testing Exclusion Criteria: * None
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What it tests

  • Carglumic Acid
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Where

5 sites
Children's National Hospital
Washington D.C., United States
University of South Florida
Tampa, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States
Riley Children's Hospital
Indianapolis, United States
Icahn School of Medicine at Mt. Sinai
New York, United States
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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