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

Primary Purpose

Hematologic Diseases

Status

Recruiting

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

Allogeneic Stem Cell Transplant

CliniMACS TCR α/β Reagent Kit and CliniMACS CD19

About this trial

This is an interventional treatment trial for Hematologic Diseases

Eligibility Criteria

1 Month - 60 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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 benfit from HSCT;
A minimum genotypic identical match of 5/10 is required;
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;
Lansky/Karnofsky score > 50;
Signed written informed consent;
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:

Pregnant or lactating females;
Greater than Grade II acute GvHD or severe, unmanaged chronic extensive GvHD due to a previous allograft at the time of inclusion;
Dysfunction of liver (ALT/AST > 10 times upper normal value, or direct bilirubin > 3 times upper normal value), or unmanageable dysfunction of renal function;
Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 30%);
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 sponsor-investigator, would place the patient at increased risk during participation in the study.

Sites / Locations

Lucile Packard Children's HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Stem Cell Transplant

Arm Description

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.

Outcomes

Primary Outcome Measures

Number of participants with successful engraftment at day 42 after HSCT

Number of participants with grade II-IV acute GvHD at day 100 after HSCT

Number of participants with grade III-IV acute GvHD at day 100 after HSCT

Secondary Outcome Measures

Number of participants with chronic GVHD at 1 year after HSCT

Leukemia-free survival at 1 year after HSCT

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

Leukemia-free survival at 2 years after HSCT

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

Number of participants with secondary graft failure at 1 year after HSCT

Number of participants with secondary graft failure at 2 years after HSCT

Full Information

NCT ID

NCT04249830

First Posted

January 29, 2020

Last Updated

February 2, 2022

Sponsor

Alice Bertaina

1. Study Identification

Unique Protocol Identification Number

NCT04249830

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2022

Overall Recruitment Status

Recruiting

Study Start Date

February 1, 2020 (Actual)

Primary Completion Date

February 2025 (Anticipated)

Study Completion Date

February 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Alice Bertaina

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Yes

Device Product Not Approved or Cleared by U.S. FDA

Yes

Data Monitoring Committee

Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hematologic Diseases

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

50 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Stem Cell Transplant

Arm Type

Experimental

Arm Description

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.

Intervention Type

Biological

Intervention Name(s)

Allogeneic Stem Cell Transplant

Intervention Description

The allogeneic stem cell transplant involves transferring the stem cells from a healthy person (donor) to the participant via infusion.

Intervention Type

Device

Intervention Name(s)

CliniMACS TCR α/β Reagent Kit and CliniMACS CD19

Intervention Description

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 Measure Information:

Title

Number of participants with successful engraftment at day 42 after HSCT

Time Frame

Day 42 after HSCT

Title

Number of participants with grade II-IV acute GvHD at day 100 after HSCT

Time Frame

Through Day 100 after HSCT

Title

Number of participants with grade III-IV acute GvHD at day 100 after HSCT

Time Frame

Through Day 100 after HSCT

Secondary Outcome Measure Information:

Title

Number of participants with chronic GVHD at 1 year after HSCT

Time Frame

1 year after HSCT

Title

Leukemia-free survival at 1 year after HSCT

Description

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

Time Frame

1 year after HSCT

Title

Leukemia-free survival at 2 years after HSCT

Description

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

Time Frame

2 years after HSCT

Title

Number of participants with secondary graft failure at 1 year after HSCT

Time Frame

1 year after HSCT

Title

Number of participants with secondary graft failure at 2 years after HSCT

Time Frame

2 years after HSCT

10. Eligibility

Sex

All

Minimum Age & Unit of Time

1 Month

Maximum Age & Unit of Time

60 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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 benfit from HSCT; A minimum genotypic identical match of 5/10 is required; 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; Lansky/Karnofsky score > 50; Signed written informed consent; 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: Pregnant or lactating females; Greater than Grade II acute GvHD or severe, unmanaged chronic extensive GvHD due to a previous allograft at the time of inclusion; Dysfunction of liver (ALT/AST > 10 times upper normal value, or direct bilirubin > 3 times upper normal value), or unmanageable dysfunction of renal function; Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 30%); 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 sponsor-investigator, would place the patient at increased risk during participation in the study.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Alice Bertaina, MD, PhD

Organizational Affiliation

Associate Professor of Pediatrics, Stem Cell Transplantation

Official's Role

Principal Investigator

Facility Information:

Facility Name

Lucile Packard Children's Hospital

City

Palo Alto

State/Province

California

ZIP/Postal Code

94306

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

SCGT Clinical Trials Program

Phone

650-497-2447

DL-SCTIntakeCoordinators@stanfordchildrens.org

First Name & Middle Initial & Last Name & Degree

David Shyr, MD

12. IPD Sharing Statement

Plan to Share IPD

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

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