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

Not Applicable

Recruiting

• Conditions: Hematologic Diseases
• Interventions: Biological: Allogeneic Stem Cell Transplant; Device: CliniMACS TCR α/β Reagent Kit and CliniMACS CD19

• Registration Number: NCT04249830
• Lead Sponsor: Alice Bertaina

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-01-29
• Study First Submitted QC: 2020-01-29
• Study First Posted: 2020-01-31
• Study Start: 2020-02-01
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-17
• Last Update Posted: 2024-04-19
• Primary Completion: 2025-02
• Study Completion: 2025-02

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 154

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Stem Cell Transplant -Malignant	Allogeneic Stem Cell Transplant	The participant with a malignancy 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.
Stem Cell Transplant - Non-Malignant	CliniMACS TCR α/β Reagent Kit and CliniMACS CD19	The participant with a non-malignant disease 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.
Stem Cell Transplant - Non-Malignant	Allogeneic Stem Cell Transplant	The participant with a non-malignant disease 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.
Stem Cell Transplant -Malignant	CliniMACS TCR α/β Reagent Kit and CliniMACS CD19	The participant with a malignancy 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.

Primary Outcome Measures

Name	Time	Method
Number of participants with grade II-IV acute GvHD at day 100 after HSCT	Through Day 100 after HSCT

Secondary Outcome Measures

Name	Time	Method
Number of participants with secondary graft failure at 1 year after HSCT	1 year after HSCT
Number of participants with secondary graft failure at 2 years after HSCT	2 years after HSCT
Number of participants with chronic GVHD at 1 year after HSCT	1 year after HSCT
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.
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.
Number of participants with successful engraftment at day 42 after HSCT	Day 42 after HSCT
Number of participants with grade III-IV acute GvHD at day 100 after HSCT	Through Day 100 after HSCT

Trial Locations

Locations (1)

Lucile Packard Children's Hospital; 🇺🇸 Palo Alto, California, United States