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Rare disease mystery unraveled: 5-Year study tracks atypical morquio a

NCT ID NCT03204370

Knowledge-focused Sponsor: GOIZET Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study followed 7 adults with a milder form of Morquio A disease for 5 years to learn how the condition changes over time. Researchers measured walking ability and other health factors to better understand the disease and improve future treatment evaluations. The goal was to gather knowledge, not to test a new treatment.

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

7 people

The number who actually took part.

Started

Feb 2018

Finished

Sep 2025

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

An unique large series of 9 adult patients (aged from 18 to 55 years) followed in a single expert center for metabolic disorders.

Ages

18 years and older

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * MPS4A affected patients * Height more than 1 m (atypical phenotypes) * Treatment by ERT or not * Followed in our expert center * Having signed an inform consent form * Being affiliated to a health insurance system Exclusion Criteria: * Patients affected by another disease * Patients refusing to participate to the study

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Conditions

The condition(s) this trial relates to.

Mucopolysaccharidosis IV mucopolysaccharidosis type 4A

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Mucopolysaccharidosis iv A

Contacts and locations

Locations

  • Rheumatology department - Bordeaux University Hospital

    Bordeaux, Aquitaine, 33076, France

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