Status:
COMPLETED
Neurocognitive Outcomes and Quality of Life in Adults With Maple Syrup Urine Disease (MSUD)
Lead Sponsor:
Children's Hospital of Philadelphia
Conditions:
Maple Syrup Urine Disease
Eligibility:
All Genders
21-80 years
Brief Summary
Maple Syrup Urine Disease (MSUD) is a disorder of protein metabolism that leads to neurological differences. It is an exciting time where people diagnosed with MSUD are living longer. The investigator...
Eligibility Criteria
Inclusion
- Inclusion Criteria for Index Subjects:
- Males or females 21 years and older
- Diagnosis of MSUD
- Ability to communicate verbally or using augmentative communication effectively to participate in appropriate neurocognitive testing
- Inclusion Criteria for Comparison Subjects:
- Males or females 21 years and older
- Sibling or acquaintance of index subject
- Ability to communicate verbally or using augmentative communication effectively to participate in appropriate neurocognitive testing
- Exclusion Criteria for all subjects:
- Inability to communicate verbally or using augmentative communication effectively to participate in appropriate neurocognitive testing
- Subjects or guardians or who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
- Subjects or guardians who do not have internet access
- Subjects who do not speak English as a primary language
Exclusion
Key Trial Info
Start Date :
June 12 2021
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
November 30 2023
Estimated Enrollment :
44 Patients enrolled
Trial Details
Trial ID
NCT04828863
Start Date
June 12 2021
End Date
November 30 2023
Last Update
June 4 2024
Active Locations (1)
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1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19143