An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
NCT ID: NCT05040178
Last Updated: 2025-04-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
20 participants
OBSERVATIONAL
2022-06-30
2032-06-30
Brief Summary
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Detailed Description
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Only available data will be collected as part of the study including developmental outcomes, details of treatment with Carbaglu® and other treatments for hyperammonemia including dietary and protein management, plasma ammonia levels, pregnancy and maternal complications, adverse effects on the developing fetus and neonate, adverse effects on the infant through first year of life.
Conditions
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Study Design
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COHORT
OTHER
Study Groups
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Male and Female Adult and Pediatric Participants
Patients treated with Carbaglu for the treatment for hyperammonemia due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA)
Carglumic Acid
Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.
Interventions
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Carglumic Acid
Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
ALL
No
Sponsors
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Target PharmaSolutions, Inc.
INDUSTRY
Recordati Rare Diseases
INDUSTRY
Responsible Party
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Principal Investigators
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William Ludlum, MD
Role: STUDY_DIRECTOR
Recordati Rare Diseases Inc.
Nicholas Ah Mew, MD
Role: PRINCIPAL_INVESTIGATOR
Children's National Research Institute
Locations
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Children's National Hospital
Washington D.C., District of Columbia, United States
University of South Florida
Tampa, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Riley Children's Hospital
Indianapolis, Indiana, United States
Icahn School of Medicine at Mt. Sinai
New York, New York, United States
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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CARBAGLU-RRDUS-PASS-0573
Identifier Type: -
Identifier Source: org_study_id
NCT05842837
Identifier Type: -
Identifier Source: nct_alias
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