Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Young Adults

Alice Bertaina

Status: Enrolling

Conditions: Hematologic Diseases

Treatments: Biological: Allogeneic Stem Cell Transplant; Device: CliniMACS TCR α/β Reagent Kit and CliniMACS CD19

Study type: Interventional

Funder types: Other

Identifiers

NCT04249830

BMT 361 - Alpha Beta IDE (Other Identifier)

IRB-53822

Details and patient eligibility

About

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.

Enrollment: 154 estimated patients

Sex: All

Ages: 1 month to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Age < 60 years and > 1 month;
2. Life expectancy > 10 weeks;
3. Patients deemed eligible for allogeneic HSCT per institutional guidelines;
4. Patients with life-threatening hematological malignancies and non-malignant disorders that could benfit from HSCT;
5. A minimum genotypic identical match of 5/10 is required;
6. 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-Cw, and HLA-DRB1;
7. Lansky/Karnofsky score > 50;
8. Signed written informed consent;
9. 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.

Exclusion criteria

1. Pregnant or lactating females;
2. Greater than Grade II acute GvHD or severe, unmanaged chronic extensive GvHD due to a previous allograft at the time of inclusion;
3. Dysfunction of liver (ALT/AST > 10 times upper normal value, or direct bilirubin > 3 times upper normal value), or unmanageable dysfunction of renal function;
4. Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 30%);
5. Current active infectious disease (including positive HIV serology or viral RNA);
6. Serious concurrent uncontrolled medical disorders;
7. Lack of patient's/parents'/guardian's informed consent;
8. Any severe concurrent disease which, in the judgement of the sponsor-investigator, would place the patient at increased risk during participation in the study.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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154 participants in 2 patient groups

Stem Cell Transplant -Malignant

Experimental group

Description:

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.

Treatment:

Biological: Allogeneic Stem Cell Transplant

Device: CliniMACS TCR α/β Reagent Kit and CliniMACS CD19

Stem Cell Transplant - Non-Malignant

Experimental group

Description:

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.

Treatment:

Biological: Allogeneic Stem Cell Transplant

Device: CliniMACS TCR α/β Reagent Kit and CliniMACS CD19