Non-invasive Functional Assessment and Pathogenesis of Morquio A

Recruiting now

Conditions studied: Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA

In brief

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.

Key facts

Study ID

NCT05284006

Run by

Nemours Children's Clinic

People needed

60

Starts

2021-05-01

Expected to finish

2027-04-30

Last updated by the study team

2026-05-13

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• No

Where it is running

• Nemours Children's Health, Delaware Valley — Wilmington, Delaware, United States (enrolling)

Full record on ClinicalTrials.gov

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