Ignite Creation Date:
2024-05-06 @ 7:17 PM
Last Modification Date:
2024-10-26 @ 3:04 PM
Study NCT ID:
NCT05968170
Status:
NOT_YET_RECRUITING
Last Update Posted:
2023-12-01
First Post:
2022-04-18
Brief Title:
TCRαβCD19 Depletion of Stem Cell Grafts for Transplant
Sponsor:
Neena Kapoor MD
Organization:
Childrens Hospital Los Angeles
Study Overview
Official Title:
A Feasibility Study Using the CliniMACS Device for T-Cell Receptor TCR αβCD19 Depleted Hematopoietic Stem Cells for Patients Undergoing Transplant
Status:
NOT_YET_RECRUITING
Status Verified Date:
2023-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The CliniMACS device is FDA-approved only for one indication CD34 selection Additional use of this device outside of this indication requires the use of feasibility studies
Children adolescents and young adults with malignant and non-malignant conditions undergoing hematopoietic stem cell transplants will have stem cells selected using alpha-betaCD19 cell depletion This is a single arm feasibility study using this processing of peripheral stem cells with alternative donor sources haploidentical mismatched matched unrelated to determine efficacy as seen by engraftment and graft-versus-host disease GVHD
Children adolescents and young adults with malignant and non-malignant conditions undergoing hematopoietic stem cell transplants will have stem cells selected using alpha-betaCD19 cell depletion This is a single arm feasibility study using this processing of peripheral stem cells with alternative donor sources haploidentical mismatched matched unrelated to determine efficacy as seen by engraftment and graft-versus-host disease GVHD
Detailed Description:
Hematopoietic stem cell transplantation HSCT is recognized as an effective cure for a wide range of diagnoses including hematologic malignancies bone marrow failure syndromes red blood cell disorders sickle cell beta thalassemia white blood cell disorders CGD and immune deficiency disorders Current therapy with allogeneic HCT from HLA-matched sibling donors has shown to be a potentially curative option for children with high-risk andor relapsed hematologic malignancies ALLAML as well as primary immune deficiency disorders PID however only 25-30 of patients have an HLA-identical matched sibling Alternative stem cell sources include matched unrelated donors MUD and unrelated cord blood UCB however the likelihood of finding an unrelated match can range between 29-79 depending on the patients ethnic background Since 2015 the CHLA Transplant and Cellular Therapy Program has performed approximately 90 ex vivo processed haploidentical transplants Greater than 80 of our patients belong to racialethnic groups with limited unrelated donor availability relying heavily on haploidentical donors This lack of matched stem cells represents a significant access disparity for underrepresented minorities with life-threatening hematologic or immunologic conditions to undergo a potentially curative HSCT
The FDA approved the use of the CliniMACS CD34 Reagent System as a Humanitarian Use Device for the prevention of GVHD in patients with acute myeloid leukemia AML in first complete remission undergoing allogeneic hematopoietic stem cell transplantation HSCT from a matched related donor The CliniMACS CD34 Reagent System decreases the risk of developing GVHD by efficiently removing donor T-cells from the graft prior to infusion by enriching CD34 blood stem cells which help to repopulate the patients immune system FDA approval was based on data from a phase II single-arm multi-center study conducted by the Blood and Marrow Transplant Clinical Trials Network that showed after an intensive myeloablative conditioning receiving a stem cell transplant from a matched related donor processed through the CliniMACS CD34 Reagent System as a GVHD prophylaxis led to a low incidence of chronic GVHD about 19 at 2 years post-transplant However removal of all cells except CD34 selected complicates immune recovery delay in CD4 cells leading to higher rates of opportunistic viral infections and transplant-related mortality
The use of CD34 selected processing has facilitated approximately 42 HSCTs in combination of TCRαβCD19 depletion at CHLA The new approach to ex vivo processing utilizes negative depletion of cells thought to be responsible for the development of aGVHD αβ TCR positive T-cells and includes simultaneous depletion of CD19 B-cells Since 2015 CHLA has conducted TCRαβCD19 depleted HSCTs successfully on several protocols including the ONC1401 KIR Study IDE16412
The FDA approved the use of the CliniMACS CD34 Reagent System as a Humanitarian Use Device for the prevention of GVHD in patients with acute myeloid leukemia AML in first complete remission undergoing allogeneic hematopoietic stem cell transplantation HSCT from a matched related donor The CliniMACS CD34 Reagent System decreases the risk of developing GVHD by efficiently removing donor T-cells from the graft prior to infusion by enriching CD34 blood stem cells which help to repopulate the patients immune system FDA approval was based on data from a phase II single-arm multi-center study conducted by the Blood and Marrow Transplant Clinical Trials Network that showed after an intensive myeloablative conditioning receiving a stem cell transplant from a matched related donor processed through the CliniMACS CD34 Reagent System as a GVHD prophylaxis led to a low incidence of chronic GVHD about 19 at 2 years post-transplant However removal of all cells except CD34 selected complicates immune recovery delay in CD4 cells leading to higher rates of opportunistic viral infections and transplant-related mortality
The use of CD34 selected processing has facilitated approximately 42 HSCTs in combination of TCRαβCD19 depletion at CHLA The new approach to ex vivo processing utilizes negative depletion of cells thought to be responsible for the development of aGVHD αβ TCR positive T-cells and includes simultaneous depletion of CD19 B-cells Since 2015 CHLA has conducted TCRαβCD19 depleted HSCTs successfully on several protocols including the ONC1401 KIR Study IDE16412
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
True
Is an Unapproved Device?:
True
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None