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

Hematologic Diseases Clinical Trial

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 Details — Status: Recruiting

Administrative data

• NCT number: NCT04249830
• Other study ID #: IRB-53822
• Secondary ID: BMT 361 - Alpha
• Status: Recruiting
• Phase: N/A
• Start date: February 1, 2020
• Est. completion date: February 2025

Study information

• Verified date: April 2024
• Source: Stanford University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 154
• Est. completion date: February 2025
• Est. primary completion date: February 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Month to 60 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hematologic Diseases

Intervention

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 a/ß 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 aß+ T cells and CD19+ B cells from HPC(A) products).

Locations

Country	Name	City	State
United States	Lucile Packard Children's Hospital	Palo Alto	California

Sponsors (1)

Lead Sponsor	Collaborator
Alice Bertaina

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with grade II-IV acute GvHD at day 100 after HSCT		Through Day 100 after HSCT
Secondary	Number of participants with chronic GVHD at 1 year after HSCT		1 year after HSCT
Secondary	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.	1 year after HSCT
Secondary	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.	2 years after HSCT
Secondary	Number of participants with secondary graft failure at 1 year after HSCT		1 year after HSCT
Secondary	Number of participants with secondary graft failure at 2 years after HSCT		2 years after HSCT
Secondary	Number of participants with grade III-IV acute GvHD at day 100 after HSCT		Through Day 100 after HSCT
Secondary	Number of participants with successful engraftment at day 42 after HSCT		Day 42 after HSCT