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NCT05284006 · ClinicalTrials.gov registry record
Non-invasive Functional Assessment and Pathogenesis of Morquio A
A clinical trial of MPS IVA and Mucopolysaccharidosis IV Type A, sponsored by Nemours Children's Clinic.
- Recruiting
- Registry status
- 60
- Enrollment target
- 1
- Study location
NCT05284006: Recruiting study of MPS IVA and Mucopolysaccharidosis IV Type A, sponsored by Nemours Children's Clinic.
NCT05284006 is a study of MPS IVA and Mucopolysaccharidosis IV Type A that is actively recruiting participants, run by Nemours Children's Clinic. The registered enrollment target is 60 participants, roughly in line with the 57-participant average among 5 other MPS IVA trials with a reported enrollment target. The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05284006, a study of MPS IVA and Mucopolysaccharidosis IV Type A, is actively recruiting participants, sponsored by Nemours Children's Clinic.
- RECRUITING
- Registry status
- 60 participants
- Enrollment target
- 1
- Study location
Study 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.
Primary Outcome
cm: The patient lies on a flat surface with knees flattened to extend the legs fully. Standing height will also be measured
Conditions Studied
Interventions
- DIAGNOSTIC_TEST Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
Study Locations (1)
Delaware
- Nemours Children's Health, Delaware Valley - Wilmington
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 60 participants |
| Start Date | 2021-05-01 |
| Est. Completion | 2026-04-30 |
What NCT05284006 shows while recruiting
NCT05284006 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 60 participants, a relatively small participant target, roughly in line with the 57-participant average among 5 other MPS IVA trials with a reported enrollment target.
The record links to 3 conditions, with MPS IVA appearing as the primary indexed condition, and to 1 intervention - of which Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc. is the first listed.
NCT05284006 reports a single indexed study location in Delaware.
Frequently Asked Questions
What is clinical trial NCT05284006 about?
NCT05284006 is a clinical study titled "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...
What is the current status of trial NCT05284006?
This trial is currently recruiting. The enrollment target is 60 participants. The study started on 2021-05-01. Estimated completion is 2026-04-30.
What conditions does trial NCT05284006 study?
This clinical trial studies the following conditions: MPS IVA, Mucopolysaccharidosis IV Type A, Morquio A Syndrome.
What interventions are being tested in trial NCT05284006?
The interventions under investigation include: Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc. (DIAGNOSTIC_TEST).
Who is sponsoring clinical trial NCT05284006?
This trial is sponsored by Nemours Children's Clinic, which has 108 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05284006 being conducted?
This trial has 1 study location across Delaware. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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