Hematologic DiseasesNCT04249830
Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Young Adults
A study testing Allogeneic Stem Cell Transplant and CliniMACS TCR α/β Reagent Kit and CliniMACS CD19 in people with Hematologic Diseases, run by Alice Bertaina at 1 site in United States.
Trial summary
The purpose of the CliniMACS® TCRαβ-Biotin System and CliniMACS® CD19 is to improve the safety and efficacy of allogeneic HLA-partially matched related or unrelated donors HSCT when no matched donors are available, to treat malignant and nonmalignant disorders for which HSCT is the recommended best available therapy. Read more about this study
- Who is it for?
- Ages 1 month to 60 years
- Conditions the study is for
- Hematologic Diseases
- What is being tested?
- Allogeneic Stem Cell Transplant and CliniMACS TCR α/β Reagent Kit and CliniMACS CD19
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 204 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Feb 2020to Dec 2030
- Phase
- Interventional
- 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 1 month to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age < 60 years and > 1 month;
- Life expectancy > 10 weeks;
- Patients deemed eligible for allogeneic HSCT per institutional guidelines;
- Patients with life-threatening hematological malignancies and non-malignant disorders that could benefit from HSCT;
- For malignant patients: i. High-risk acute lymphoblastic leukemia (ALL) in 1st complete remission (CR), ALL in 2nd CR; or ii. High-risk acute myeloid leukemia (AML) in 1st CR, AML in 2nd CR; or iii. Childhood Myelodysplastic Syndrome (MDS) with low blasts (cMDS-LB) or Childhood MDS with increased blasts (cMDS-IB); or iv. Juvenile myelomonocytic leukemia (JMML); or v. Mixed-phenotype acute leukemia (MPAL); or vi. Non-Hodgkin lymphomas in 2nd CR; or vii. Other hematologic malignancies in 1st or 2nd CR eligible for stem cell transplantation per institutional standard b. Patients with non-malignant disorders receiving first HSCT: i. using mis-matched donors, due to the absence of suitable HLA identical sibling or HLA phenotypically identical relative; or ii. whose disease put them at increased risk of graft rejection or GvHD (e.g., Fanconi Anemia, STAT1 gain of function) and therefore can benefit from receiving alpha beta depleted HSCT using as a donor either an HLA identical sibling or an HLA phenotypically identical (10/10 matched) donor;
- A minimum genotypic identical match of 5/10 is required;
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- The donor and recipient must be identical, as determined by high resolution typing, in at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-C, HLA-DQB1 and HLA-DRB1;
- Lansky/Karnofsky score > 50; the Karnofsky Scale will be used in subjects ≥ 16 years of age, and the Lansky Scale will be used for those < 16 years of age.
- All subjects ≥ 18 years of age must be able to give informed consent or adults lacking capacity to consent must have a legally authorized representative (LAR) available to provide consent. For subjects <18 years old their legal authorized representative (LAR) (i.e. parent or guardian) must give informed consent. Pediatric subjects will be included in age appropriate discussion and written assent will be obtained for those > 7 years of age, when appropriate
- Male and female subjects of childbearing potential must agree to use an effective means of birth control to avoid pregnancy throughout the transplant procedure, while on immunosuppression, and if the subject experiences any chronic GvHD.
Who cannot join
- Pregnant or lactating females;
- Has received a prior allogenic HSCT;
- Secondary MDS or AML or treatment related MDS or AML;
- Dysfunction of liver (ALT/AST > 10 times upper normal value, or direct bilirubin > 3 times upper normal value),
- Serum creatinine > 1.5 times ULN (for patients not on dialysis) or unmanageable dysfunction of renal function while undergoing dialysis (for patients on dialysis);
- Severe cardiovascular disease (congestive heart failure or left ventricular ejection fraction < 30%);
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- Current active infectious disease (including positive HIV serology or viral RNA);
- Serious concurrent uncontrolled medical disorders;
- Lack of patient's/parents'/guardian's informed consent;
- Any severe concurrent disease which, in the judgement of the PI, would place the patient at increased risk during participation in the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Lucile Packard Children's Hospital
Palo Alto, California, 94306
SCGT Clinical Trials Program, (650) 497-2447, DL-SCTIntakeCoordinators@stanfordchildrens.org
Tami John, MD
About this study
Official title: Allogeneic Hematopoietic Stem Cell Transplantation From an HLA-partially Matched Related or Unrelated Donor After TCR αβ+T Cells/CD19+ B Cell Depletion in Children and Young Adults Affected by Malignant or Non-Malignant Hematological Disorders
The purpose of the CliniMACS® TCRαβ-Biotin System and CliniMACS® CD19 is to improve the safety and efficacy of allogeneic HLA-partially matched related or unrelated donors HSCT when no matched donors are available, to treat malignant and nonmalignant disorders for which HSCT is the recommended best available therapy. Initially this device will be used in a single-center, open-label, single-arm, phase II clinical trial to evaluate the efficacy of haploidentical PBSC grafts depleted of TCRα/β+ and CD19+ cells using the CliniMACS® TCRαβ/CD19 System in children and adults with hematological and non-hematological malignancies.
- Conditions
- Hematologic Diseases
- Being tested
- Allogeneic Stem Cell Transplant, CliniMACS TCR α/β Reagent Kit and CliniMACS CD19
- Medical subject headings
- Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Stem Cell TransplantBiological
The allogeneic stem cell transplant involves transferring the stem cells from a healthy person (donor) to the participant via infusion.
CliniMACS TCR α/β Reagent Kit and CliniMACS CD19Device
The CliniMACS™system can be used to selectively enrich or reduce specific cell populations based on the magnetic cell selection (MACS) technology developed by Miltenyi Biotec. Cell mixtures can be separated in a magnetic field using one or more immunomagnetic- labeled antibodies specific for the cell types of interest (e.g.TCR αβ+ T cells and CD19+ B cells from HPC(A) products).
| Study group | Role | What they receive |
|---|---|---|
| Stem Cell Transplant -Malignant | Experimental | The participant with a malignancy will undergo a stem cell transplant using donor cells that have been manipulated through an investigational device. Participants will be followed for outcomes for two years. Receives: Biological: Allogeneic Stem Cell Transplant; Device: CliniMACS TCR α/β Reagent Kit and CliniMACS CD19 |
| Stem Cell Transplant - Non-Malignant | Experimental | The participant with a non-malignant disease will undergo a stem cell transplant using donor cells that have been manipulated through an investigational device. Participants will be followed for outcomes for two years. Receives: Biological: Allogeneic Stem Cell Transplant; Device: CliniMACS TCR α/β Reagent Kit and CliniMACS CD19 |
| Stem Cell Transplant - Compassionate | Other | Patients with malignant or non-malignant disorders who do not qualify for experimental arms but who may still benefit from participation in this study may be enrolled in this arm. Receives: Biological: Allogeneic Stem Cell Transplant; Device: CliniMACS TCR α/β Reagent Kit and CliniMACS CD19 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with grade II-IV acute GvHD after HSCT
Measured: Through Day 100 after HSCT
Secondary outcomes (5)
Leukemia-free survival after HSCT
Leukemia-free survival defined as the time of enrollment to disease relapse or death from any cause.
Measured: 1 and 2 years after HSCT
Number of participants with secondary graft failure at after HSCT
Measured: 1 and 2 year after HSCT
Number of participants with grade III-IV acute GvHD after HSCT
Measured: Through Day 100 after HSCT
Incidence rate of primary graft failure after HSCT
Measured: Day 42 after HSCT
Incidence of moderate and severe chronic GvHD after HSCT
Measured: 1 year after HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2020
- Study start1 Feb 2020
- Primary completionDec 2028 (estimated)
- Study completionDec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic DiseasesStudies of the same condition, open and under way.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 204 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alice Bertaina
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Alice Bertaina, Stanford University
- Investigators
- David Shyr, MD, Principal investigator, Clinical Associate Professor, Pediatrics, Stem Cell Transplantation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04249830
- Sponsor's protocol ID
- IRB-53822
- Other IDs
- BMT 361 - Alpha Beta IDE
- Status last verified
- May 2025
- First submitted
- 29 Jan 2020
- Last updated
- 29 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Hematopoietic Stem Cell Transplantation From an HLA-partially Matched Related or Unrelated Donor After TCR αβ+T Cells/CD19+ B Cell Depletion in Children and Young Adults Affected by Malignant or Non-Malignant Hematological Disorders
- ClinicalTrials.gov ID
- NCT04249830
- Acronym / sponsor ID
- IRB-53822
- Conditions
- Hematologic Diseases
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 204 participants (estimated)
- Lead sponsor
- Alice BertainaUniversity, hospital or other
- Study start
- 1 Feb 2020 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2030 (estimated)
- First posted
- 31 Jan 2020 (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.
The purpose of the CliniMACS® TCRαβ-Biotin System and CliniMACS® CD19 is to improve the safety and efficacy of allogeneic HLA-partially matched related or unrelated donors HSCT when no matched donors are available, to treat malignant and nonmalignant disorders for which HSCT is the recommended best available therapy. Initially this device will be used in a single-center, open-label, single-arm, phase II clinical trial to evaluate the efficacy of haploidentical PBSC grafts depleted of TCRα/β+ and CD19+ cells using the CliniMACS® TCRαβ/CD19 System in children and adults with hematological and non-hematological malignancies.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Stem Cell Transplant -Malignant | Experimental | Biological: Allogeneic Stem Cell Transplant, Device: CliniMACS TCR α/β Reagent Kit and CliniMACS CD19The participant with a malignancy will undergo a stem cell transplant using donor cells that have been manipulated through an investigational device. Participants will be followed for outcomes for two years. |
| Stem Cell Transplant - Non-Malignant | Experimental | Biological: Allogeneic Stem Cell Transplant, Device: CliniMACS TCR α/β Reagent Kit and CliniMACS CD19The participant with a non-malignant disease will undergo a stem cell transplant using donor cells that have been manipulated through an investigational device. Participants will be followed for outcomes for two years. |
| Stem Cell Transplant - Compassionate | Other | Biological: Allogeneic Stem Cell Transplant, Device: CliniMACS TCR α/β Reagent Kit and CliniMACS CD19Patients with malignant or non-malignant disorders who do not qualify for experimental arms but who may still benefit from participation in this study may be enrolled in this arm. |
Who may qualify
- Ages
- 1 month to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Age < 60 years and > 1 month;
- Life expectancy > 10 weeks;
- Patients deemed eligible for allogeneic HSCT per institutional guidelines;
- Patients with life-threatening hematological malignancies and non-malignant disorders that could benefit from HSCT;
- A minimum genotypic identical match of 5/10 is required;
Key exclusion criteria (10 listed)
- Pregnant or lactating females;
- Has received a prior allogenic HSCT;
- Secondary MDS or AML or treatment related MDS or AML;
- Dysfunction of liver (ALT/AST > 10 times upper normal value, or direct bilirubin > 3 times upper normal value),
- Serum creatinine > 1.5 times ULN (for patients not on dialysis) or unmanageable dysfunction of renal function while undergoing dialysis (for patients on dialysis);
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 |
|---|---|---|
| Allogeneic Stem Cell TransplantThe allogeneic stem cell transplant involves transferring the stem cells from a healthy person (donor) to the participant via infusion. | Biological | Stem Cell Transplant - Compassionate, Stem Cell Transplant - Non-Malignant, Stem Cell Transplant -Malignant |
| CliniMACS TCR α/β Reagent Kit and CliniMACS CD19The CliniMACS™system can be used to selectively enrich or reduce specific cell populations based on the magnetic cell selection (MACS) technology developed by Miltenyi Biotec. Cell mixtures can be separated in a magnetic field using one or more immunomagnetic- labeled antibodies specific for the cell types of interest (e.g.TCR αβ+ T cells and CD19+ B cells from HPC(A) products). | Device | Stem Cell Transplant - Compassionate, Stem Cell Transplant - Non-Malignant, Stem Cell Transplant -Malignant |
Primary outcome
Number of participants with grade II-IV acute GvHD after HSCT
Time frame: Through Day 100 after HSCT
5 secondary outcomes
Leukemia-free survival after HSCT
Time frame: 1 and 2 years after HSCT
Number of participants with secondary graft failure at after HSCT
Time frame: 1 and 2 year after HSCT
Number of participants with grade III-IV acute GvHD after HSCT
Time frame: Through Day 100 after HSCT
Incidence rate of primary graft failure after HSCT
Time frame: Day 42 after HSCT
Incidence of moderate and severe chronic GvHD after HSCT
Time frame: 1 year after HSCT
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 May 2025.
- Individual participant dataSharing planned: No
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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.
Lucile Packard Children's Hospital
Palo Alto, United States
David Shyr, MD principal investigator
Clinical Associate Professor, Pediatrics, Stem Cell Transplantation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Allogeneic Stem Cell Transplant and CliniMACS TCR α/β Reagent Kit and CliniMACS CD19 in people with Hematologic Diseases, run by Alice Bertaina 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 10 inclusion and 10 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 May 2025. 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.
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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with grade II-IV acute GvHD after HSCT (time frame: Through Day 100 after HSCT). 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 May 2025.