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Scientists launch Largest-Ever study to unravel Cystinosis's Long-Term effects

NCT ID NCT07680751

Knowledge-focused Sponsor: Institut National de la Santé Et de la Recherche Médicale, France 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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 02, 2026 · Last updated Sep 18, 2026 · Updated 2 times

Summary

This study follows 250 people with cystinosis, a rare genetic disorder that causes a buildup of the amino acid cystine, leading to damage in the kidneys, eyes, and other organs. Researchers will track how the disease progresses over time, including kidney function, eye problems, and quality of life, while also collecting blood samples for future research. The goal is to better understand the long-term course of the disease under current treatments and identify factors that may predict complications.

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 study could reveal better ways to manage cystinosis and identify biomarkers that predict complications, potentially improving long-term care.
What could go wrong
As an observational study, it does not test a new treatment, so it cannot directly lead to a cure or new therapy. Results may take years to influence clinical practice.

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 250 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Mar 2028

An estimate. End dates often move.

Lead sponsor

A government agency

The lead sponsor is a government body.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Patients with a confirmed diagnosis of cystinosis followed in European expert reference centers are included in this multicenter observational cohort. The study population includes both prevalent patients already under follow-up and incident patients identified during the recruitment period. Included patients are European patients receiving standard of care and followed in France, Belgium, Italy, Germany, Spain, and the Netherlands. Both pediatric and adult patients are eligible. No intervention is assigned as part of the study. Approximately 250 patients are expected to be enrolled and followed longitudinally.

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

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

Inclusion Criteria: * Confirmed diagnosis of cystinosis based on leukocyte cystine measurement, presence of corneal cystine crystals, and/or molecular genetic diagnosis * Signed informed consent obtained from the patient or legal representative Exclusion Criteria: * Patients unable to provide informed consent or without a legal representative when required * No other specific exclusion criteria; patients with associated diseases may be included

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Conditions

The condition(s) this trial relates to.

cystinosis

As listed by the trial registrant

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

Cystinosis

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

  • AP-HP_ Hôpital Charles Foix

    Ivry-sur-Seine, Île-de-France Region, 94200, France

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