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Non-invasive Functional Assessment and Pathogenesis of Morquio A
Sponsor: Nemours Children's Clinic
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 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.
Official title: Non-invasive Functional Assessment and Pathogenesis of Morquio A (NIFAMA)
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
60
Start Date
2021-05-01
Completion Date
2026-04-30
Last Updated
2025-05-14
Healthy Volunteers
No
Interventions
Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
This study includes 15 major assessments: clinical assessment procedures; anthropometric measurements; activity of daily living and quality-of-life questionnaires; gait kinematics and kinetics analysis; pulmonary function tests (PFT); skeletal radiographs and dual-energy x-ray absorptiometry (DXA); MRI in cervical spine, temporal bones, and hip; computed tomography angiography (CTA) for tracheal obstruction; CT for temporal bones; anesthetic encounters; joint mobility; hearing function; biochemical analyses; and pathological analyses.
Locations (1)
Nemours Children's Health, Delaware Valley
Wilmington, Delaware, United States