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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04249830
Recruitment Status : Recruiting
First Posted : January 31, 2020
Last Update Posted : January 31, 2020
Sponsor:
Information provided by (Responsible Party):
Alice Bertaina, Stanford University

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
Hematologic Diseases Biological: Allogeneic Stem Cell Transplant Device: CliniMACS TCR α/β Reagent Kit and CliniMACS CD19 Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated 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
Estimated Study Start Date : February 2020
Estimated Primary Completion Date : February 2025
Estimated Study Completion Date : February 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Blood Disorders

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).




Primary Outcome Measures :
  1. Number of participants with successful engraftment at day 42 after HSCT [ Time Frame: Day 42 after HSCT ]
  2. Number of participants with grade II-IV acute GvHD at day 100 after HSCT [ Time Frame: Through Day 100 after HSCT ]
  3. Number of participants with grade III-IV acute GvHD at day 100 after HSCT [ Time Frame: Through Day 100 after HSCT ]

Secondary Outcome Measures :
  1. Number of participants with chronic GVHD at 1 year after HSCT [ Time Frame: 1 year after HSCT ]
  2. Leukemia-free survival at 1 year after HSCT [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 1 year after HSCT ]
  5. Number of participants with secondary graft failure at 2 years after HSCT [ Time Frame: 2 years after HSCT ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   1 Month to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04249830


Locations
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United States, California
Lucile Packard Children's Hospital Recruiting
Palo Alto, California, United States, 94306
Contact: Study Team    650-497-2447    scgt_clinical_trials_office@lists.stanford.edu   
Sub-Investigator: David Shyr, MD         
Sub-Investigator: Alison Holzer-Speed, PhD         
Sponsors and Collaborators
Alice Bertaina
Investigators
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Principal Investigator: Alice Bertaina, MD, PhD Associate Professor of Pediatrics, Stem Cell Transplantation
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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 ( Other Identifier: Stanford University )
First Posted: January 31, 2020    Key Record Dates
Last Update Posted: January 31, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: Yes
Additional relevant MeSH terms:
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Hematologic Diseases