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New hope for rare genetic disorder: tailored chemo before stem cell transplant

NCT ID NCT07316595

Disease control Sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests whether a lower dose of the chemotherapy drug treosulfan, given before a stem cell transplant, can help people with Nijmegen breakage syndrome. The condition causes immune problems and a high risk of cancer. The trial will enroll 24 patients, giving a lower dose to those without cancer and a higher dose to those with cancer, aiming to improve survival and reduce complications.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Treosulfan (a chemotherapy drug)
What this could lead to
If successful, this could make stem cell transplants safer and more effective for people with Nijmegen breakage syndrome, potentially reducing the risk of graft rejection and cancer relapse.
What could go wrong
This is a small, early-phase trial with only 24 participants, so results may not apply to everyone. The drug is still experimental for this condition, and there are risks of serious side effects like graft-versus-host disease or organ toxicity.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 24 people

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

Expected to start

Dec 2025

An estimate. Start dates often move.

Expected to finish

Dec 2030

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

Up to 21 years

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: * genetically confirmed Nijmegen breakage syndrome * availability of informed consent for study participation, signed by the patient (ages 14 to 21) and/or their legal representative (ages 0 to 18). * absence of contraindications to HSCT based on the patient's somatic status. Exclusion Criteria: * other DNA repair deficiency syndromes (both specified and unspecified)

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Conditions

The condition(s) this trial relates to.

Nijmegen breakage syndrome

As listed by the trial registrant

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

HSCT Nijmegen breakage syndrome Treosulfan based conditioning

How to take part

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

  1. 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 ↗

  2. 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.

More trials for these conditions

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