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New stem cell method aims to protect kids with rare immune disease

NCT ID NCT02737384

Disease control 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
The trial has finished. Results may not be published yet.
Stopped early This study
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This status has not been confirmed recently, so it may be out of date.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 tested a way to improve stem cell transplants for children with combined immunodeficiency (CID), a condition where the immune system doesn't work properly. The approach removed certain naive cells from the donor stem cells to try to prevent graft-versus-host disease (a serious complication) while keeping the donor's memory T cells to fight infections. Only 4 children were enrolled before the study ended early, so results are limited.

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

4 people

The number who actually took part.

Started

Jun 2016

Finished

Oct 2017

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

12 months to 18 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: * Patient from 12 months to 18 years * Combined immunodeficiencies with known molecular diagnosis or if unknown, corresponding of p-CID study's definition * Hematopoietic stem cell Transplantation planned with one of the following donors : * sibling with 1 or 2 HLA antigens mismatch * parent 10/10 or 9/10 identical * unrelated donor: 10/10 or 9/10 identical * Consent form signed by the child's legal guardian * Patient using effectiveness contraception during this trial * Affiliated or beneficiary of a health insurance regimen Exclusion Criteria: * Wiskott-Aldrich syndrome * Ongoing pregnancy * Positive HIV PCR * Contraindication for hematopoetic stem cell transplantation * Geno-identical donor in the siblings * hematopoetic stem cell transplantation antecedent

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Conditions

The condition(s) this trial relates to.

combined immunodeficiency

As listed by the trial registrant

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

Combined immunodeficiencies

Contacts and locations

Locations

  • Hôpital Necker-Enfants Malades

    Paris, Paris, 75015, France