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New stem cell approach could fix immune systems in kids

NCT ID NCT02990819

Disease control Sponsor: Children's Hospital of Philadelphia 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Phase II trial tests a stem cell transplant from unrelated or partially matched family donors for children and young adults with severe immune deficiencies. The stem cells are specially processed to remove certain immune cells, aiming to reduce complications. The goal is to see if this approach leads to stable donor cell engraftment and improved survival.

What this could mean

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

Active substance
stem cell transplant with alpha/beta T and CD19+ cell depletion
What this could lead to
If successful, this could provide a safer way to restore immune function in children with life-threatening immune disorders using donor stem cells.
What could go wrong
This is an early Phase II trial with only 60 participants, so results may not apply to everyone. Risks include graft failure, graft-versus-host disease, and infection.

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 60 people

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

Start date

Dec 2016

Expected to finish

Dec 2027

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 25 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: 1. Ages 0-25 years at time of enrollment 2. Diseases: * Immunodeficiencies for which allogeneic hematopoietic stem cell transplant is indicated, including severe combined immunodeficiencies, immunodeficiency polyendocrinopathy X-linked syndrome (IPEX), X-linked lymphoproliferative disease, chronic granulomatous disease, Wiskott-Aldrich syndrome (WAS), hyperIgM, and other life-threatening immunodeficiencies. * Immune dysregulation syndromes, including refractory or recurrent hemophagocytic lymphohistiocytosis, hemophagocytic lymphohistiocytosis (HLH) with genetic mutations, refractory multisystemic Langerhans cell histiocytosis, other macrophage activating syndrome (MAS) refractory to standard therapy. 3. Clinical status * Lansky or Karnofsky performance \>=60 * Organ Function: 1. Serum creatinine \<1.5 x upper limit of normal for age Hepatic: ALT \<=250; AST \<=350 2. Cardiac shortening fraction \>=27% 3. Bilirubin \<2.5x normal (unless elevation due to Gilberts disease). 4. No active untreated infection 4. Signed informed consent 5. No HLA matched related donor available. 6. Females of childbearing potential must have negative pregnancy test. Exclusion Criteria: * Uncontrolled bacterial, viral or fungal infections * HLA matched related or unrelated donor able to donate mobilized peripheral stem cells. * Pregnant Females * Matched related donor available for bone marrow donation Donors Selection Criteria: * Donor selection will comply with 21 CFR 1271 * Unrelated donor matched or up to one antigen mismatch as per National Marrow Donor Program (NMDP). * Haploidentical parent or sibling able to undergo mobilization for peripheral stem cell collection. Maternal donor preferred over paternal donor if both equally haploidentical. * Children's Hospital of Philadelphia (CHOP) Blood and Marrow Transplant (BMT) procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. * Unrelated donor identified through the National Marrow Donor Program (NMDP) and fulfills the NMDP criteria for donation. Unrelated donor willing and able to undergo mobilization of peripheral stem cells and apheresis

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Conditions

The condition(s) this trial relates to.

chronic granulomatous disease hemophagocytic syndrome hyper-IgM syndrome immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome immunodeficiency disease inborn error of immunity Langerhans cell histiocytosis macrophage activation 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.

Immune dysregulation syndromes Immunodeficiencies

Contacts and locations

Locations

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

More trials for these conditions

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