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Gentler transplant shows promise for kids with blood diseases

NCT ID NCT01050855

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 22, 2026 · Updated 3 times

Summary

This study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer side effects than standard transplants. Researchers are tracking how well the donor cells take and overall survival.

What this could mean

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

Active substance
Reduced intensity conditioning regimen (Campath, Fludarabine, Melphalan, Busulfan, Cyclosporine, Cellcept)
What this could lead to
If successful, this could offer a safer transplant option for children with non-malignant blood disorders, reducing side effects and improving long-term survival.
What could go wrong
This is a mid-stage trial with only 75 participants, so results may not apply to everyone. The regimen still carries risks like graft failure or 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

56 people

The number who actually took part.

Started

Jan 2008

Finished

Jun 2025

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

6 months 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. Age \>6 months- 25 years 2. Diseases eligible for Distal Alemtuzumab: * Immunodysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome * Sickle cell disease (Neurologic: history of stroke or any neurologic defect lasting \>24 hours accompanied by infarct on MRI; or abnormal transcranial Doppler, or abnormal MRI with cerebral vasculature stenosis. OR minimum 2 episodes of acute chest syndrome in preceding 2 year period OR history 3 or more severe pain events/year in the 2 years prior to transplant.) * Thalassemia major * Bone marrow failure, including Kostmann's, amegakaryocytic thrombocytopenia, Blackfan-Diamond syndrome 3. Diseases eligible for Intermediate Alemtuzumab * Hemophagocytic lymphohistiocytosis other macrophage activation syndromes, severe Langerhans histiocytosis * Severe combined immune deficiency, adenosine deaminase deficiency, common variable immunodeficiency * Wiskott-Aldrich syndrome 4. Organ criteria: * Cardiac: Echocardiogram shortening fraction \>27% * Pulmonary: for those with pulmonary function tests: DLCO/VA \>40% If DLCO/VA is not reported this is considered inability to perform the test. In this case Pulse Ox and Respiratory Exam will be used as evaluation criteria. * Renal: Serum creatinine \<1.5 x upper limit of normal for age * Hepatic:; ALT and AST \<5 x upper limit of normal * Infection: No active infections. Exclusion criteria 1. Uncontrolled bacterial, fungal or viral infections. 2. Bare lymphocyte syndrome (MHC class II deficiency)

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Conditions

The condition(s) this trial relates to.

beta thalassemia bone marrow failure syndrome common variable immunodeficiency disease hemoglobinopathy hemophagocytic syndrome immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome immunodeficiency disease inborn error of immunity inherited hemoglobinopathy Langerhans cell histiocytosis macrophage activation syndrome severe combined immunodeficiency severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency sickle cell disease Wiskott-Aldrich syndrome

As listed by the trial registrant

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

Hemoglobinopathies Immunodeficiencies Non malignant diseases

Contacts and locations

Locations

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

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