A Non-Interventional Post-Authorization Study of Carbaglu for the Treatment of Hyperammonemia Due to MMA and PA
Launched by TARGET PHARMASOLUTIONS, INC. · Apr 24, 2023
Trial Information
Current as of July 01, 2025
Enrolling by invitation
Keywords
ClinConnect Summary
No description provided
Gender
All
Eligibility criteria
- Inclusion Criteria:
- • Provide a signed and dated informed consent/assent form
- • Receive treatment with Carbaglu
- Have an established diagnosis of PA or MMA defined as at least 1 of the following:
- • Confirmation by molecular genetic testing of gene mutations responsible for PA or MMA
- • 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
- • 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)
- Exclusion Criteria:
- • None
About Target Pharmasolutions, Inc.
Target Pharmasolutions, Inc. is a leading clinical trial sponsor dedicated to advancing pharmaceutical research and development. With a focus on innovative solutions, the company specializes in providing comprehensive clinical trial management services that enhance the efficiency and effectiveness of drug development. Target Pharmasolutions leverages cutting-edge technology and a team of experienced professionals to ensure regulatory compliance and optimize patient engagement, ultimately facilitating the successful delivery of new therapies to market. Committed to quality and integrity, the company partners with biopharmaceutical organizations to support their clinical goals and drive transformative outcomes in healthcare.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Indianapolis, Indiana, United States
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
10 years