Non-invasive Functional Assessment and Pathogenesis of Morquio A

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 that can be applicable to all ages of patients to better understand the progress of their disease and the most serious clinical problems (cervical instability and stenosis, tracheal obstruction, hyperlaxity of joints, hip dysplasia, and small lung capacity). The outcome of this project will lead to a more precise understanding of the skeletal/pulmonary compromise and defining clinical endpoints in this disease for future clinical trials of current or developing therapies.

NCT Number	NCT05284006
Lead Sponsor	Nemours Children's Clinic Collaborators: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Conditions	Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
Enrollment	60 participants
Start Date	2021-05-01
Primary Completion	2027-04-30 (estimated)
Study Completion	2027-04-30 (estimated)
Updated on ClinicalTrials.gov	2026-05-13