An interventional study of CliniMACS® device in Graft Vs Host Disease, Graft-versus-host-disease and Acute Myeloid Leukemia, sponsored by Neena Kapoor, M.D.. Withdrawn. Open to participants aged Up to 21 Years. Per ClinicalTrials.gov, last updated 2025-07-14.
Sponsored by Neena Kapoor, M.D. · Not applicable, Interventional, and Treatment
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-beta+/CD19+ 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).
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 and/or relapsed hematologic malignancies (ALL/AML) 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 patient's 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 racial/ethnic 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 patient's 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 TCRαβ+/CD19 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 TCRαβ/CD19+ depleted HSCTs successfully on several protocols, including the ONC1401 KIR Study (IDE#16412).
806 studies on the registry are indexed under Graft vs Host Disease; 138 are open to participants now.
Browse Graft vs Host Disease studies →Neena Kapoor, M.D. is the lead sponsor of 3 studies on the registry; none are open to participants now.
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Adequate organ function (within 4 weeks of initiation of preparative regimen) as evaluated per institutional guidelines. Adequate major organ system function as demonstrated by:
Exclusion Criteria:
The test product is an αβ+/CD19+ T-cell depleted stem cell product using the CliniMACS system. The test product is given intravenously over a period of time as dictated by the final volume of the infused product (5mL/kg/hour). The target dose of CD34+ cells is 20-40 x 10\^6/kg, but a minimum of 5 x 10\^6/kg is required. The target dose of TCRαβ+ T-cells and CD19+/CD20+ T-cells is ≤ 1 x 10\^5/kg.
Device: CliniMACS® device
CliniMACS TCR αβ+/CD19+ cell depletion for related or unrelated haploidentical/mismatched/matched hematopoietic stem cell transplant patients and/or high risk GVHD patients.
Also known as: CliniMACS® CD19 reagent system, CliniMACS® depletion tubing set, CliniMACS® TCR α/β reagent system
Incidence of grade 3-4 acute and/or chronic GVHD at Day+100
Assess the cumulative incidence of severe GVHD following HSCT with TCRαβ+/CD19+ T-cell depleted grafts, as determined by the presence of Grade III-IV aGVHD and/or cGVHD by Day+100 post-HSCT.
Time frame: Day+100 post-HSCT
Incidence of engraftment at Day+30.
Assess the efficacy of TCRαβ+/CD19+ T-cell depleted HSCTs as measured by the engraftment of donor cells by Day+30 post-HSCT.
Time frame: Day+30 post-HSCT
Incidence of transplant-related mortality at 1-year post-HSCT.
Assess the efficacy of TCRαβ+/CD19+ T-cell depleted HSCTs as measured by the incidence of transplant-related mortality at 1-year post-HSCT.
Time frame: 1-Year post-HSCT
Incidence of T-cell reconstitution at Day+180 (CD4+ T-cell count > 200 and proliferation to PHA > 50% control).
Assess the immune reconstitution following TCRαβ+/CD19+ T-cell depleted HSCTs as measured by the presence of expanded T-cell numbers and function at Day+180 post-HSCT(CD4+ T-cell count \> 200 and proliferation to PHA \> 50% control).
Time frame: Day+180 post-HSCT
Incidence of microbial contamination of the infused cells product leading to possible infection in the recipient (i.e., antibiotics needed for > 48 hours).
Assess the risk of microbial contamination in TCRαβ+/CD19+ T-cell depleted products from the CliniMACS® device.
Time frame: Day+30
No study locations are listed for this record.
Plan to share: Yes — The data will be made available internally to other sub-investigators that have expressed interest. Data that will be shared must go through the Sponsor-Investigator and will be redacted (if not already done) prior to receiving the data so that no identifying information is present.
Supporting information: Study protocol, Icf
This study is withdrawn, as verified in Jul 2025. You cannot join it, but the record below documents what was studied.
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Neena Kapoor, M.D.