NCT04806347 Alpha/Beta T-cell Depleted Blood-forming Stem Cell Transplant From Related or Unrelated Donors for Blood Diseases in Children and Young Adults
| NCT ID | NCT04806347 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | University of Wisconsin, Madison |
| Condition | Blood Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 12 participants |
| Start Date | 2026-11 |
| Primary Completion | 2029-06 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 12 participants in total. It began in 2026-11 with a primary completion date of 2029-06.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
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).
Contact & Investigator
Christian Capitini, MD
PRINCIPAL INVESTIGATOR
University of Wisconsin, Madison
Frequently Asked Questions
Who can join the NCT04806347 clinical trial?
This trial is open to participants of all sexes, aged 3 Months or older, up to 25 Years, studying Blood Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04806347 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT04806347 currently recruiting?
Yes, NCT04806347 is actively recruiting participants. Contact the research team at PedsHemOncResearch@lists.wisc.edu for enrollment information.
Where is the NCT04806347 trial being conducted?
This trial is being conducted at Madison, United States.
Who is sponsoring the NCT04806347 clinical trial?
NCT04806347 is sponsored by University of Wisconsin, Madison. The principal investigator is Christian Capitini, MD at University of Wisconsin, Madison. The trial plans to enroll 12 participants.