Clinical Trials /

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

NCT04249830

Description:

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.

Related Conditions:
  • Hematopoietic and Lymphoid Malignancy
Recruiting Status:

Recruiting

Phase:

N/A

Trial Eligibility

Document

Title

  • Brief Title: Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Young Adults
  • 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

Clinical Trial IDs

  • ORG STUDY ID: IRB-53822
  • SECONDARY ID: BMT 361 - Alpha Beta IDE
  • NCT ID: NCT04249830

Conditions

  • Hematologic Diseases

Interventions

DrugSynonymsArms
Allogeneic Stem Cell TransplantStem Cell Transplant

Purpose

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.

Trial Arms

NameTypeDescriptionInterventions
Stem Cell TransplantExperimentalThe participant 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.
  • Allogeneic Stem Cell Transplant

Eligibility Criteria

        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.
      
Maximum Eligible Age:60 Years
Minimum Eligible Age:1 Month
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Number of participants with successful engraftment at day 42 after HSCT
Time Frame:Day 42 after HSCT
Safety Issue:
Description:

Secondary Outcome Measures

Measure:Number of participants with chronic GVHD at 1 year after HSCT
Time Frame:1 year after HSCT
Safety Issue:
Description:
Measure:Leukemia-free survival at 1 year after HSCT
Time Frame:1 year after HSCT
Safety Issue:
Description:Leukemia-free survival defined as the time of enrollment to disease relapse or death from any cause.
Measure:Leukemia-free survival at 2 years after HSCT
Time Frame:2 years after HSCT
Safety Issue:
Description:Leukemia-free survival defined as the time of enrollment to disease relapse or death from any cause.
Measure:Number of participants with secondary graft failure at 1 year after HSCT
Time Frame:1 year after HSCT
Safety Issue:
Description:
Measure:Number of participants with secondary graft failure at 2 years after HSCT
Time Frame:2 years after HSCT
Safety Issue:
Description:

Details

Phase:N/A
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Alice Bertaina

Last Updated

January 29, 2020