Status:
RECRUITING
Non-invasive Functional Assessment and Pathogenesis of Morquio A
Lead Sponsor:
Nemours Children's Clinic
Collaborating Sponsors:
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Conditions:
Mucopolysaccharidosis IV Type A
Morquio A Syndrome
Eligibility:
All Genders
Brief Summary
Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establish a non-invasive objective assessment t...
Detailed Description
Mucopolysaccharidosis IVA (MPS IVA, Morquio A Disease) is a rare autosomal recessive disorder caused by a deficiency of the lysosomal enzyme, N-acetylgalactosamine 6-sulfate sulfatase (GALNS). GALNS c...
Eligibility Criteria
Inclusion
- Patients affected by MPS IVA. The diagnosis of MPS IVA is confirmed by deficient enzyme activity of \< 5% of normal activity level as measured in plasma or leukocytes.
Exclusion
- No
Key Trial Info
Start Date :
May 1 2021
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
April 30 2026
Estimated Enrollment :
60 Patients enrolled
Trial Details
Trial ID
NCT05284006
Start Date
May 1 2021
End Date
April 30 2026
Last Update
May 14 2025
Active Locations (1)
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1
Nemours Children's Health, Delaware Valley
Wilmington, Delaware, United States, 19803