Alpha/Beta T-cell Depleted Blood-forming Stem Cell Transplant for Children and Young Adults

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1 trial evaluates the safety and feasibility of delivering TCRαβ+/CD19+ depleted hematopoietic stem cell grafts obtained from closely matched related or unrelated donors to treat non-malignant blood diseases in patients up to young adulthood. The graft engineering uses the Miltenyi CliniMACS system to selectively remove αβ T cells and B cells while preserving other immune cell subsets to support engraftment and immune reconstitution. Enrollment targets children and young adults without an HLA-identical sibling or readily available matched donor, including haploidentical or closely matched unrelated donors. The study focuses on reducing graft-versus-host disease while maintaining graft function and immune defense, within an initial safety framework at a single institution. Participation hinges on specific disease criteria and donor availability, with close monitoring for adverse events and engraftment outcomes.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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)
* Age ≤ 25 years (except patients with hemoglobinopathies)
* Life Expectancy ≥ 3 months
* Karnofsky (patients \> 16 years)/Lansky (patients ≤ 16 years) index ≥ 60
* Organ Function Requirements

  * Renal Function

    * Creatinine clearance or radioisotope Glomerular Filtration Rate (GFR) greater than or equal to 60 ml/min/1.73m\^2
  * Liver Function

    * Total bilirubin \< 3 mg/dL
    * Alanine aminotransferase (ALT)/ Serum glutamic-pyruvic transaminase; synonymous with ALT (SCPT) ≤ 3 x Upper Limit of Normal (ULN) for age
  * Cardiac Function

    * Ejection fraction of \> 40% by Multiple gated acquisition scan (MUGA) or echocardiogram
  * Pulmonary Function

    * No evidence of dyspnea at rest
    * No supplemental oxygen requirement
    * If measured, carbon monoxide diffusion capacity (DLCO) \> 50%
* Willing to use effective birth control method if patient is of reproductive potential
* Informed consent obtained (patient or legal representative)

Exclusion Criteria:

* 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).

Where it's running

• American Family Children's Hospital
  Madison, Wisconsin, United States Recruiting

Study contacts

• Principal investigator: Christian Capitini, MD — University of Wisconsin, Madison
• Study coordinator: Jenny Weiland
• Email: PedsHemOncResearch@lists.wisc.edu
• Phone: 608-890-8070

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.