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Milder stem cell transplant shows promise for kids with immune disorders

NCT ID NCT01821781

Disease control Sponsor: Washington University School of Medicine 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tests a stem cell transplant using a milder chemotherapy-like regimen to treat children and young adults (up to age 28) with various immune system disorders. The goal is to help the donor stem cells settle in the body with fewer side effects. The study involves 20 participants and focuses on whether the transplant works and how safe it is.

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 20 people

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

Started

Apr 2013

Expected to finish

Apr 2026

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: * \</= 28 years of age * Performance status \>/= 40 * DLCO \>/= 40% * LVEF \>/=40% or LVSF \>/=26% * Serum creatinine \< 2x ULN * Liver enzymes \</= 5x ULN * Negative pregnancy test * Suitably matched donor (6/6 matched sib UCB, 8/8 matched sib BM or PBSC, 5-6/6 matched unrelated UCB, 7-8/8 matched unrelated BM, double cord) Exclusion Criteria: * Known diagnosis of HIV I/II * Pregnant or breastfeeding * Uncontrolled invasive fungal or bacterial infections within 1 month prior to starting alemtuzumab * Uncontrolled viral infection within 1 week prior to starting alemtuzumab

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Conditions

The condition(s) this trial relates to.

autoimmune lymphoproliferative syndrome Bruton-type agammaglobulinemia Chediak-Higashi syndrome chronic granulomatous disease common variable immunodeficiency DiGeorge syndrome hemophagocytic syndrome hyper-IgM syndrome immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome immune system disorder inborn error of immunity Lymphohistiocytosis, Hemophagocytic lymphoproliferative syndrome severe combined immunodeficiency Wiskott-Aldrich syndrome X-linked lymphoproliferative syndrome

As listed by the trial registrant

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

Autoimmune lymphoproliferative syndrome Chediak-Higashi syndrome Chronic granulomatous disease Common variable immune deficiency DiGeorge syndrome Hemophagocytic lymphohistiocytosis Hyper-IgM Immune deficiency disorders Immune dysregulatory disorders IPEX Severe combined immunodeficiency Wiskott-Aldrich syndrome X-linked agammaglobulinemia X-linked lymphoproliferative syndrome

Contacts and locations

Locations

  • Washington University

    St Louis, Missouri, 63110, United States

More trials for these conditions

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