Blood DiseasePhase 1NCT04806347
Alpha/Beta T-cell Depleted Blood-forming Stem Cell Transplant From Related or Unrelated Donors for Blood Diseases in Children and Young Adults
A Phase 1 study testing TCRαβ+/CD19+ depleted Hematopoietic stem cell (HSC) graft and CliniMACS® System in people with Blood Disease, run by University of Wisconsin, Madison at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 months to 25 years
- Conditions the study is for
- Blood Disease
- What is being tested?
- TCRαβ+/CD19+ depleted Hematopoietic stem cell (HSC) graft and CliniMACS® System
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2026to Jun 2030
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 months to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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:
Show all 42Show fewer
- 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)
Who cannot join
- 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).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Celeste Matsushima contact
United States1 site, 1 recruiting
- Recruiting
American Family Children's Hospital
Madison, Wisconsin, 53792
About this study
Official title: TCRαβ+ and CD19+ Depleted Hematopoietic Stem Cell Transplant From Closely Matched Unrelated Donors or Haploidentical Related Donors for Hematologic Diseases in Children and Young Adults
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.
Read the detailed description
This single institution, phase I clinical trial will determine the safety and feasibility of employing T-cell receptor (TCR) αβ+ and CD19+ (Cluster of Differentiation) depleted hematopoietic stem cell transplantation (HSCT) using peripheral blood stem cells (PBMC) from closely matched unrelated donors or haploidentical donors to treat non-malignant hematologic diseases in children and young adults. Allogeneic hematopoietic stem cell transplantation has become a curative option for children and adolescents with a variety of otherwise fatal conditions. To reduce the incidence and severity of graft-versus-host disease (GVHD) associated with allogeneic hematopoietic stem cell transplantation, donor grafts are depleted of T cells, either using CD34+ selection or CD3+/CD19+ depletion of grafts. However, these selection processes also deplete the graft of protective cell subsets, such as γδ T cells, natural killer (NK) cells, monocytes and dendritic cells, which play important roles in the immune response to infectious agents. Moreover, the presence of NK cells and γδ T cells in donor grafts is associated with more rapid immune reconstitution after HSCT transplantation. In order to retain these protective immune cell subsets, this trial will use a novel, highly selective graft engineering process using the Miltenyi CliniMACS system that selectively depletes αβ-T cells and B cells that are responsible for GVHD and Epstein Barr Virus (EBV)-related post-transplantation lymphoproliferative disorder, respectively. Prior to transplantation, patients will be treated with a conditioning regimen, specific for the original disorder.
The primary objective of this study is evaluation of the safety and feasibility of HSCT using TCRαβ+/CD19+ depleted hematopoietic stem cells to treat non-malignant hematologic diseases. This will be assessed by evaluating the incidence of graft failure, grade III-IV acute GVHD and chronic GVHD and TRM.
Secondary objectives include the evaluation of immune reconstitution and incidence of post-transplant infections, adverse events, serious adverse events, overall and disease-free survival and the efficiency of graft processing by the CliniMACS System.
- Conditions
- Blood Disease
- Being tested
- TCRαβ+/CD19+ depleted Hematopoietic stem cell (HSC) graft, CliniMACS® System
- Medical subject headings
- Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TCRαβ+/CD19+ depleted Hematopoietic stem cell (HSC) graftBiological
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® SystemDevice
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.
| Study group | Role | What they receive |
|---|---|---|
| Treatment arm | Experimental | 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. Receives: Biological: TCRαβ+/CD19+ depleted Hematopoietic stem cell (HSC) graft; Device: CliniMACS® System |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of grade III-IV acute graft-versus-host disease (GVHD)
Acute GVHD will be assessed and graded according to the Keystone Consensus Criteria for staging and grading of acute graft-versus-host disease.
Measured: 100 days post transplantation
Incidence of extensive chronic GVHD
Chronic GVHD will be assessed according to the current CIBMTR (Center for International Blood and Marrow Transplant Research) manual reflecting a grading system published by Sullivan KM (Sullivan 1981).
Measured: up to 2 years
Incidence of graft failure
Graft failure - defined as failure to achieve ANC > 500 /µL at Day +28 or initial neutrophil engraftment followed by a decline in ANC < 500 /µL that is unresponsive to growth factor therapy (secondary graft failure).
Measured: up to 2 years after graft
Incidence of Treatment Related Mortality (TRM)
TRM - defined as death from any cause other than disease progression.
Measured: Day +100 post-HSCT
Secondary outcomes (15)
Time to neutrophil engraftment
The time to neutrophil engraftment is defined as the post-transplant day that is the first of 3 consecutive days with an absolute neutrophil count (ANC) of >500/µL as assessed by CBC.
Measured: up to 28 days following HSCT
Time to platelet engraftment
The time to platelet engraftment is defined as the post-transplant day that is the first of 3 consecutive days with a platelet count ≥ 20,000/µL as assessed by complete blood count (CBC), without platelet support (transfusion) for 7 days.
Measured: up to 28 days following HSCT
Percentage donor chimerism using Short Tandem Repeat (STR)
Short tandem repeat (STR) analysis will provide the percentage donor chimerism at specific time points post-transplant.
Measured: up to 12 months following HSCT
Kinetics of lymphocyte reconstitution via immunophenotyping using flow cytometry
Lymphocyte reconstitution will be assessed at specific time points post-HSCT, expressed as the percentage of white blood cells comprised of T, B and NK cell subsets.
Measured: up to 12 months following HSCT
CliniMACS system efficiency: Percentage of viable CD34+ cells recovered after the TCRαβ+ and CD19+ depletion procedure
Measured: up to 12 months following HSCT
CliniMACS system efficiency: log depletion value for CD19/CD20+ B cells after the TCRαβ+ and CD19+ depletion procedure
Measured: Day 0
CliniMACS system efficiency: log depletion value of TCRαβ+ T cells after the TCRαβ+ and CD19+ depletion procedure
Measured: Day 0
CliniMACS system efficiency: number of viable blood cell subsets after the TCRαβ+ and CD19+ depletion procedure
Number of viable CD34+ blood stem cells, CD20+ B cells, CD3-CD56+ NK cells, TCRαβ+ T cells, and TCRγδ+ T cells in the HSC graft after the TCRαβ+ and CD19+ depletion procedure
Measured: Day 0
Correlation between GVHD incidence and donor killer-cell immunoglobulin-like receptor (KIR) haplotype content
Measured: up to 2 years
Correlation between GVHD incidence and killer-cell immunoglobulin-like receptor (KIR)/KIR-ligand mismatch between donor and recipient.
Measured: up to 2 years
Event free survival
An event is defined as death, graft failure or stable mixed chimerism with disease recurrence.
Measured: up to 1 years
Overall survival (OS)
Measured: up to 2 years
Incidence of symptomatic bacterial/fungal and viral reactivation requiring therapy
The incidence of infections (symptomatic bacterial/fungal and viral reactivation requiring therapy) will be used as an additional safety measure
Measured: up to 1 year
Number of participants with adverse events related to infusion of the HSC graft
Measured: Day 0
Incidence of serious adverse events
Measured: up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2021
- Study startNov 2026 (estimated)
- Primary completionJun 2029 (estimated)
- Study completionJun 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Blood DiseaseStudies of the same condition, open and under way.
- More trials run by University of Wisconsin, MadisonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Wisconsin, Madison
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Christian Capitini, MD, Principal investigator, University of Wisconsin, MadisonJacques Galipeau, MD, Study director, University of Wisconsin, Madison
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04806347
- Sponsor's protocol ID
- 2020-1251
- Other IDs
- 2020-1251, A536755, Protocol Version 6, NCI-2021-02128, UW19113
- Status last verified
- Sep 2026
- First submitted
- 2 Mar 2021
- Last updated
- 29 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- Yes: The Principal Investigator, Sponsor and funding institutions (if applicable) will ensure that all mechanisms used to share data include proper plans and safeguards to protect the rights and privacy of participants who participate in this research.
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- TCRαβ+ and CD19+ Depleted Hematopoietic Stem Cell Transplant From Closely Matched Unrelated Donors or Haploidentical Related Donors for Hematologic Diseases in Children and Young Adults
- ClinicalTrials.gov ID
- NCT04806347
- Acronym / sponsor ID
- 2020-1251
- Conditions
- Blood Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- University of Wisconsin, MadisonUniversity, hospital or other
- Study start
- Nov 2026 (estimated)
- Primary completion
- Jun 2029 (estimated)
- Study completion
- Jun 2030 (estimated)
- First posted
- 19 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This single institution, phase I clinical trial will determine the safety and feasibility of employing T-cell receptor (TCR) αβ+ and CD19+ (Cluster of Differentiation) depleted hematopoietic stem cell transplantation (HSCT) using peripheral blood stem cells (PBMC) from closely matched unrelated donors or haploidentical donors to treat non-malignant hematologic diseases in children and young adults. Allogeneic hematopoietic stem cell transplantation has become a curative option for children and adolescents with a variety of otherwise fatal conditions. To reduce the incidence and severity of graft-versus-host disease (GVHD) associated with allogeneic hematopoietic stem cell transplantation, donor grafts are depleted of T cells, either using CD34+ selection or CD3+/CD19+ depletion of grafts. However, these selection processes also deplete the graft of protective cell subsets, such as γδ T cells, natural killer (NK) cells, monocytes and dendritic cells, which play important roles in the immune response to infectious agents. Moreover, the presence of NK cells and γδ T cells in donor grafts is associated with more rapid immune reconstitution after HSCT transplantation. In order to retain these protective immune cell subsets, this trial will use a novel, highly selective graft engineering process using the Miltenyi CliniMACS system that selectively depletes αβ-T cells and B cells that are responsible for GVHD and Epstein Barr Virus (EBV)-related post-transplantation lymphoproliferative disorder, respectively. Prior to transplantation, patients will be treated with a conditioning regimen, specific for the original disorder.
The primary objective of this study is evaluation of the safety and feasibility of HSCT using TCRαβ+/CD19+ depleted hematopoietic stem cells to treat non-malignant hematologic diseases. This will be assessed by evaluating the incidence of graft failure, grade III-IV acute GVHD and chronic GVHD and TRM.
Secondary objectives include the evaluation of immune reconstitution and incidence of post-transplant infections, adverse events, serious adverse events, overall and disease-free survival and the efficiency of graft processing by the CliniMACS System.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment arm | Experimental | Biological: TCRαβ+/CD19+ depleted Hematopoietic stem cell (HSC) graft, Device: CliniMACS® SystemParticipants 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. |
Who may qualify
- Ages
- 3 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (42 listed)
- 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
Key exclusion criteria (5 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TCRαβ+/CD19+ depleted Hematopoietic stem cell (HSC) graftAfter 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. | Biological | Treatment arm |
| CliniMACS® SystemThe 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. | Device | Treatment arm |
Primary outcome
Incidence of grade III-IV acute graft-versus-host disease (GVHD)
Time frame: 100 days post transplantation
Acute GVHD will be assessed and graded according to the Keystone Consensus Criteria for staging and grading of acute graft-versus-host disease.
Incidence of extensive chronic GVHD
Time frame: up to 2 years
Chronic GVHD will be assessed according to the current CIBMTR (Center for International Blood and Marrow Transplant Research) manual reflecting a grading system published by Sullivan KM (Sullivan 1981).
Incidence of graft failure
Time frame: up to 2 years after graft
Graft failure - defined as failure to achieve ANC > 500 /µL at Day +28 or initial neutrophil engraftment followed by a decline in ANC < 500 /µL that is unresponsive to growth factor therapy (secondary graft failure).
Incidence of Treatment Related Mortality (TRM)
Time frame: Day +100 post-HSCT
TRM - defined as death from any cause other than disease progression.
15 secondary outcomes
Time to neutrophil engraftment
Time frame: up to 28 days following HSCT
Time to platelet engraftment
Time frame: up to 28 days following HSCT
Percentage donor chimerism using Short Tandem Repeat (STR)
Time frame: up to 12 months following HSCT
Kinetics of lymphocyte reconstitution via immunophenotyping using flow cytometry
Time frame: up to 12 months following HSCT
CliniMACS system efficiency: Percentage of viable CD34+ cells recovered after the TCRαβ+ and CD19+ depletion procedure
Time frame: up to 12 months following HSCT
CliniMACS system efficiency: log depletion value for CD19/CD20+ B cells after the TCRαβ+ and CD19+ depletion procedure
Time frame: Day 0
CliniMACS system efficiency: log depletion value of TCRαβ+ T cells after the TCRαβ+ and CD19+ depletion procedure
Time frame: Day 0
CliniMACS system efficiency: number of viable blood cell subsets after the TCRαβ+ and CD19+ depletion procedure
Time frame: Day 0
Correlation between GVHD incidence and donor killer-cell immunoglobulin-like receptor (KIR) haplotype content
Time frame: up to 2 years
Correlation between GVHD incidence and killer-cell immunoglobulin-like receptor (KIR)/KIR-ligand mismatch between donor and recipient.
Time frame: up to 2 years
Event free survival
Time frame: up to 1 years
Overall survival (OS)
Time frame: up to 2 years
Incidence of symptomatic bacterial/fungal and viral reactivation requiring therapy
Time frame: up to 1 year
Number of participants with adverse events related to infusion of the HSC graft
Time frame: Day 0
Incidence of serious adverse events
Time frame: up to 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2026.
- Individual participant dataSharing planned: Yes. The Principal Investigator, Sponsor and funding institutions (if applicable) will ensure that all mechanisms used to share data include proper plans and safeguards to protect the rights and privacy of participants who participate in this research.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Celeste Matsushima contact
Christian Capitini, MD principal investigator
University of Wisconsin, Madison
Jacques Galipeau, MD study director
University of Wisconsin, Madison
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TCRαβ+/CD19+ depleted Hematopoietic stem cell (HSC) graft and CliniMACS® System in people with Blood Disease, run by University of Wisconsin, Madison at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 42 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jenny Weiland (608-890-8070, PedsHemOncResearch@lists.wisc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of grade III-IV acute graft-versus-host disease (GVHD) (time frame: 100 days post transplantation). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Oct 2026.