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Scientists hunt for clues to predict cancer in people with rare gene mutation

NCT ID NCT05983991

Knowledge-focused Sponsor: Institut Claudius Regaud Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 follows 150 people with a GATA2 gene mutation to learn what factors predict if they will develop serious blood cancers like MDS or AML. Researchers will collect blood and bone marrow samples over 5 years and also review past data from 100 other patients. The goal is to better understand the disease, 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.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

About 150 people

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

Started

Dec 2023

Expected to finish

Dec 2033

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.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

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: 1. All subject, at any age, with a germline GATA2 mutation. 2. Patient followed in the center within a standard of care procedure or clinical trial. 3. Signed written informed consent. For minor patients: patient assent and legal guardian(s) written informed consent obtained before inclusion in the study and prior performance of any study-related procedure. 4. For French patients: patient affiliated to a Social Health Insurance. Exclusion Criteria: 1. GATA2 somatic mutation. 2. Any psychological, familial, geographic or social situation, according to the judgment of investigator, potentially preventing the provision of informed consent or compliance to study procedure. 3. Person who has forfeited his/her freedom by administrative or legal award or who is under legal protection, with the exception of persons under curatorship who may be included in the study.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia Myelodysplastic Syndromes

As listed by the trial registrant

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

GATA2 gene mutation

How to take part

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

  1. The places running it

    23 sites. 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

  • CHRU - Lille

    RECRUITING

    Lille, France

  • CHRU Nancy Hôpital d'enfants

    RECRUITING

    Vandœuvre-lès-Nancy, France

  • CHRU de Tours

    NOT_YET_RECRUITING

    Tours, 37044, France

  • CHU Angers

    RECRUITING

    Angers, France

  • CHU Arnaud de Villeneuve

    RECRUITING

    Montpellier, France

  • CHU Bordeaux

    RECRUITING

    Bordeaux, France

  • CHU Dijon

    RECRUITING

    Dijon, France

  • CHU Estaing

    RECRUITING

    Clermont-Ferrand, France

  • CHU Hôpitaux de Rouen - Charles Nicolle

    RECRUITING

    Rouen, France

  • CHU La Timone

    RECRUITING

    Marseille, France

  • CHU Nantes

    RECRUITING

    Nantes, France

  • CHU Purpan - Hôpital des Enfants

    RECRUITING

    Toulouse, France

  • CHU Rennes

    RECRUITING

    Rennes, France

  • CHU Saint-Etienne

    RECRUITING

    Saint-Priest-en-Jarez, France

  • CHU Strasbourg

    RECRUITING

    Strasbourg, France

  • CHU de Grenoble

    NOT_YET_RECRUITING

    Grenoble, 38043, France

  • CHU de Nice

    NOT_YET_RECRUITING

    Nice, 06202, France

  • Hôpital Armand Trousseau

    RECRUITING

    Paris, France

  • Hôpital Necker

    NOT_YET_RECRUITING

    Sèvres, 75015, France

  • Hôpital Robert Debré

    RECRUITING

    Paris, France

  • Hôpital Saint-Louis

    RECRUITING

    Paris, France

  • IHOP Lyon

    RECRUITING

    Lyon, France

  • Institut Universitaire Du Cancer Toulouse - Oncopole (Iuct-O)

    RECRUITING

    Toulouse, France