Recruiting

Phase 1

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

Sponsor:

University of Wisconsin, Madison

Code:

NCT04806347

Conditions

Blood Disease

Eligibility Criteria

Sex: All

Age: 0 - 25

Healthy Volunteers: Not accepted

Interventions

TCRαβ+/CD19+ depleted Hematopoietic stem cell (HSC) graft

CliniMACS® System

Study Details

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.

Conditions

Blood Disease

Study ID

NCT04806347

Start date

Oct, 2026

Status verified date

Sep, 2026

Completion date

Jun, 2030

Anticipated

Primary completion date

Jun, 2029

Anticipated

Eligibility Criteria

Sex: All

Age: 0 - 25

Healthy Volunteers: Not accepted

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).

Study Design

Enrollment

12 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment arm

Participants will undergo a conditioning regimen, specific for the original disease, After that peripheral blood stem cell transplant from a haploidentical donor or closely matched unrelated donor, depleted of TCRαβ+ and CD19+ cells using the CliniMACS TCR α/β-biotin and CD19 Systems will be administered intravenously on Day 0 to all participants.

Interventions

TCRαβ+/CD19+ depleted Hematopoietic stem cell (HSC) graft

After undergoing a disease specific conditioning regimen (standard of care), participants will receive peripheral blood stem cell transplant from a haploidentical donor or closely matched unrelated donor, depleted of TCR αβ+ and CD19+ cells using the CliniMACS TCR α/β-biotin and CD19 Systems.

CliniMACS® System

The CliniMACS Cell Selection System is based on magnetic-activated cell sorting mechanism. The CliniMACS device is a powerful tool for the isolation of many cell types from heterogeneous cell mixtures.

Primary outcome measure

Incidence of grade III-IV acute graft-versus-host disease (GVHD) [ Time Frame: 100 days post transplantation ]
Incidence of extensive chronic GVHD [ Time Frame: up to 2 years ]
Incidence of graft failure [ Time Frame: up to 2 years after graft ]
Incidence of Treatment Related Mortality (TRM) [ Time Frame: Day +100 post-HSCT ]

Central Contacts and Locations

Central contacts

Jenny Weiland

608-890-8070 PedsHemOncResearch@lists.wisc.edu

Celeste Matsushima

608-890-8069

Locations

American Family Children's Hospital

Recruiting

Madison, Wisconsin, United States, 53792

More Information

Sponsor

University of Wisconsin, Madison

Last update posted

Oct 2, 2026

Last verified

Sep, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-03. This information was provided to ClinicalTrials.gov by University of Wisconsin, Madison on 2026-10-02. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.