Alpha/Beta T-cell Depleted Blood-forming Stem Cell Transplant From Related or Unrelated Donors for Blood Diseases in Children and Young Adults

Starting soon · Phase 1

Conditions studied: Blood Disease

In brief

This study is being done to see if the investigators can take peripheral blood stem cells from either an adult family member or a closely matched unrelated donor, and run them through a special lab instrument to remove alpha/beta T cells and B cells and then give them to the patient to treat disease. This is an experimental way of doing a hematopoietic stem cell transplant (HSCT). The investigators want to see if the new stem cells will grow without bad graft vs. host disease (GVHD). This treatment approach is experimental in the United States.

Key facts

Study ID

NCT04806347

Run by

University of Wisconsin, Madison

People needed

12

Starts

2026-08-01

Expected to finish

2030-06-01

Last updated by the study team

2026-07-23

Who can join

Age: 0 and older, up to 25. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• No Human leukocyte antigen (HLA) identical sibling available AND
• NO HLA matched unrelated donor available OR urgent need of HSCT precludes time necessary to search for suitable HLA matched unrelated donor AND
• Haploidentical donor OR closely matched unrelated donor available and willing to undergo mobilization and apheresis
• If subject has genetically confirmed inherited bone marrow failure, related donor must be evaluated for this disorder and testing must be negative.
• If subject has sickle cell disease, donor must be unaffected or have only sickle cell trait
• Patient must be diagnosed with one of the following diseases or disorders:
• Hemoglobinopathies
• Sickle Cell Disease for patients ≤ 21 years of age for whom hydroxyurea has been trialed for at least six months, and failed
• Thalassemia Major for patients ≤ 21 years of age
• Acquired Bone Marrow Failure Syndromes
• Paroxysmal Nocturnal Hemoglobinuria with bone marrow failure
• Myelodysplastic Syndromes (lower risk)
• Severe acquired aplastic anemia
• Inherited Bone Marrow Failure Syndromes
• Fanconi Anemia
• Diamond Blackfan Anemia
• Dyskeratosis Congenita and related telomere disorders
• Congenital Thrombocytopenia Syndromes
• Severe Congenital Neutropenia
• Shwachman-Diamond Syndrome
• Inborn Errors of Immunity (IEI) or Primary Immune Regulatory Disorders (PIRD)
• Wiskott-Aldrich syndrome (WAS)
• Chronic granulomatous disease (CGD)
• Severe combined immunodeficiency (SCID)
• Primary hemophagocytic lymphohistiocytosis (HLH)

You may not qualify if…

• Pregnant
• HIV infection
• Uncontrolled, serious active infection at screening
• Significant serious intercurrent illnesses
• Enrollment in any other treatment study that would interfere with the endpoints of this study according to judgement of Principal Investigator (or PI designee).

Full record on ClinicalTrials.gov

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