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

Sponsor
Alice Bertaina (Other)
Overall Status
Recruiting
CT.gov ID
NCT04249830
Collaborator
(none)
50
Enrollment
1
Location
1
Arm
60
Anticipated Duration (Months)
0.8
Patients Per Site Per Month

Study Details

Study Description

Brief 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. 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.

Condition or Disease Intervention/Treatment Phase
  • Biological: Allogeneic Stem Cell Transplant
  • Device: CliniMACS TCR α/β Reagent Kit and CliniMACS CD19
 N/A

Study Design

Study Type:
Interventional
Anticipated Enrollment :
50 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
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
Actual Study Start Date :
Feb 1, 2020
Anticipated Primary Completion Date :
Feb 1, 2025
Anticipated Study Completion Date :
Feb 1, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: Stem Cell Transplant

The 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.

Biological: Allogeneic Stem Cell Transplant
The allogeneic stem cell transplant involves transferring the stem cells from a healthy person (donor) to the participant via infusion.

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

Outcome Measures

Primary Outcome Measures

  1. Number of participants with successful engraftment at day 42 after HSCT [Day 42 after HSCT]

  2. Number of participants with grade II-IV acute GvHD at day 100 after HSCT [Through Day 100 after HSCT]

  3. Number of participants with grade III-IV acute GvHD at day 100 after HSCT [Through Day 100 after HSCT]

Secondary Outcome Measures

  1. Number of participants with chronic GVHD at 1 year after HSCT [1 year after HSCT]

  2. Leukemia-free survival at 1 year after HSCT [1 year after HSCT]

    Leukemia-free survival defined as the time of enrollment to disease relapse or death from any cause.

  3. Leukemia-free survival at 2 years after HSCT [2 years after HSCT]

    Leukemia-free survival defined as the time of enrollment to disease relapse or death from any cause.

  4. Number of participants with secondary graft failure at 1 year after HSCT [1 year after HSCT]

  5. Number of participants with secondary graft failure at 2 years after HSCT [2 years after HSCT]

Eligibility Criteria

Criteria

Ages Eligible for Study:
1 Month to 60 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Lucile Packard Children's Hospital Palo Alto California United States 94306

Sponsors and Collaborators

  • Alice Bertaina

Investigators

  • Principal Investigator: Alice Bertaina, MD, PhD, Associate Professor of Pediatrics, Stem Cell Transplantation

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Alice Bertaina, Associate Professor of Pediatrics, Stanford University
ClinicalTrials.gov Identifier:
NCT04249830
Other Study ID Numbers:
  • IRB-53822
  • BMT 361 - Alpha Beta IDE
First Posted:
Jan 31, 2020
Last Update Posted:
Feb 4, 2022
Last Verified:
Feb 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
Yes
Additional relevant MeSH terms:
Hematologic Diseases

Study Results

No Results Posted as of Feb 4, 2022