Actively Recruiting
Non-invasive Functional Assessment and Pathogenesis of Morquio A
Led by Nemours Children's Clinic · Updated on 2026-05-13
60
Participants Needed
1
Research Sites
312 weeks
Total Duration
On this page
Sponsors
N
Nemours Children's Clinic
Lead Sponsor
E
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Non-invasive Functional Assessment and Pathogenesis of Morquio A
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients diagnosed with MPS IVA confirmed by enzyme activity less than 5% of normal in plasma or leukocytes
You will not qualify if you...
- None
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Nemours Children's Health, Delaware Valley
Wilmington, Delaware, United States, 19803
Actively Recruiting
Research Team
S
Shunji Tomatsu, MD PhD
CONTACT
G
Greg Stets
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here