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Rare disease study maps muscle decline in cystinosis patients

NCT ID NCT05545774

Knowledge-focused Sponsor: Assistance Publique - Hôpitaux de Paris 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study looked at how cystinosis affects muscles, breathing, and swallowing in 20 teenagers and adults. Researchers measured motor function, strength, and walking ability over time. The goal was to better understand the disease's impact on the body, 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

20 people

The number who actually took part.

Started

Sep 2022

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

Male and female, age ≥ 16 years, with confirmed diagnosis of cystinosis and presenting at least one motor deficiency, are eligibles for this study.

Ages

16 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: * Patients aged ≥ 16 years; * Genetically confirmed diagnosis of cystinosis; * Presenting motor deficiency of at least one muscle; * Covered by health insurance. Exclusion Criteria: * Patient refusal; * Foreign patients under AME health schema; * Patients under legal protection; * Pregnancy or breast-feeding.

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Conditions

The condition(s) this trial relates to.

cystinosis Deglutition Disorders neuromuscular disease Respiratory Aspiration

As listed by the trial registrant

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

Cystinosis

Contacts and locations

Locations

  • Department of Nephrology and Transplantation Centre de référence des Maladies Rénales Héréditaires de l'Enfant et de l'Adulte, Necker-Enfants Malades University Hospital, APHP

    Paris, France

  • Neurology Department, Raymond-Poincaré hospital, APHP

    Garches, 92380, France

More trials for these conditions

Other studies related to the condition(s) this trial covers.