New study aims to decode rare bone disease without surgery
NCT ID NCT05284006
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study is for children with Morquio A, a rare genetic disease that affects bones and breathing. Researchers will use safe, non-invasive tests like MRI, X-rays, hearing tests, and walking analysis to track how the disease changes over time. The goal is to better understand the disease and find better ways to measure it in future treatment studies.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- What this could lead to
- If successful, this could lead to better ways to track Morquio A progression and define clear endpoints for future treatment trials.
- What could go wrong
- This is an observational study, not a treatment trial. It will not directly improve symptoms or cure the disease. Results may take years to influence clinical care.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
-
About 60 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
May 2021
- Expected to finish
-
Apr 2027
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
The studies proposed here will involve a minimum of 60 subjects who have the biochemical defect of GALNS. There is no limitation in the age range. All patients will be included, whether ambulatory or not, with presence or absence of planned surgical operation, and whether received ERT. The study population will consist of male and female patients with the diagnosis of MPS IVA, also known as Morquio A disease. Before any study procedures, all potential subjects and their parents/legal guardians will be informed about the purpose and conduct of the study. They will be prospectively followed every 18 months at Nemours Children's Health, Delaware Valley.
- Ages
-
Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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. Exclusion Criteria: * No
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Morquio A syndrome are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The places running it
1 site. The list below names each one and where it is.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
-
Nemours Children's Health, Delaware Valley
RECRUITINGWilmington, Delaware, 19803, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.