Description:
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.
Title
- 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
Clinical Trial IDs
- ORG STUDY ID:
IRB-53822
- SECONDARY ID:
BMT 361 - Alpha Beta IDE
- NCT ID:
NCT04249830
Conditions
Interventions
Drug | Synonyms | Arms |
---|
Allogeneic Stem Cell Transplant | | Stem Cell Transplant |
Purpose
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.
Trial Arms
Name | Type | Description | Interventions |
---|
Stem Cell Transplant | Experimental | 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. | - Allogeneic Stem Cell Transplant
|
Eligibility 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.
Maximum Eligible Age: | 60 Years |
Minimum Eligible Age: | 1 Month |
Eligible Gender: | All |
Healthy Volunteers: | No |
Primary Outcome Measures
Measure: | Number of participants with successful engraftment at day 42 after HSCT |
Time Frame: | Day 42 after HSCT |
Safety Issue: | |
Description: | |
Secondary Outcome Measures
Measure: | Number of participants with chronic GVHD at 1 year after HSCT |
Time Frame: | 1 year after HSCT |
Safety Issue: | |
Description: | |
Measure: | Leukemia-free survival at 1 year after HSCT |
Time Frame: | 1 year after HSCT |
Safety Issue: | |
Description: | Leukemia-free survival defined as the time of enrollment to disease relapse or death from any cause. |
Measure: | Leukemia-free survival at 2 years after HSCT |
Time Frame: | 2 years after HSCT |
Safety Issue: | |
Description: | Leukemia-free survival defined as the time of enrollment to disease relapse or death from any cause. |
Measure: | Number of participants with secondary graft failure at 1 year after HSCT |
Time Frame: | 1 year after HSCT |
Safety Issue: | |
Description: | |
Measure: | Number of participants with secondary graft failure at 2 years after HSCT |
Time Frame: | 2 years after HSCT |
Safety Issue: | |
Description: | |
Details
Phase: | N/A |
Primary Purpose: | Interventional |
Overall Status: | Recruiting |
Lead Sponsor: | Alice Bertaina |
Last Updated
July 15, 2021