CD45RA-depleted CD19-CAR T Cell Consolidation After TCRαβ+/CD19 B Cell-depleted Haploidentical Hematopoietic Cell Transplantation for Relapsed/Refractory CD19+ ALL and Lymphoma

Purpose

The purpose of this study is to learn more about newer methods of transplanting blood cells donated by a partially matched family member to children with high-risk CD19 positive leukemia ALL. Primary Objective: - To assess the safety and feasibility of combining CD19-CAR(Mem) T cells after TCRαβ+/CD19 depleted haploidentical donor transplantation for pediatric patients with relapsed/refractory CD19+ B-cell malignancies. Secondary Objectives: - To estimate 1-year post-transplant overall survival, event-free survival, and GVHD-free relapse-free survival (GRFS). - To estimate cumulative incidence of engraftment, acute and chronic GVHD, and immune-related adverse events, including CRS and ICANS.

Conditions

  • Relapsed Pediatric ALL
  • Hematopoietic Cell Transplantation
  • Hematologic Malignancy

Eligibility

Eligible Ages
Under 21 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

Recipient - Age less than or equal to 21 years - High risk hematologic malignancy where allogeneic transplantation is the current standard of care. This includes (but is not limited to): - High risk CD19+ B cell ALL in CR1 or CR2 - Any CD19+ B-cell ALL in CR3 or subsequent - If prior CNS leukemia, it must be treated and in CNS CR - Left ventricular ejection fraction > 40%, or shortening fraction ≥ 25% - Creatinine clearance (CrCl) or glomerular filtration rate (GFR) ≥ 50 ml/min/1.73m2 - Forced vital capacity (FVC) ≥ 50% of predicted value; or pulse oximetry ≥ 92% on room air if patient is unable to perform pulmonary function testing - Karnofsky or Lansky (age dependent) performance score ≥ 50 (See APPENDIX A) - Bilirubin ≤ 3 times the upper limit of normal for age - Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age Donor - At least single haplotype matched (≥ 4 of 8) family member - At least 18 years of age - HIV negative - If sexually active, agreement to use birth control until 2 weeks after completion of the mobilization and apheresis procedure - Regarding donation eligibility, is identified as either: - Completed the process of donor eligibility determination as outlined in 21 CFR 1271 and agency guidance; OR - Does not meet 21 CFR 1271 eligibility requirements, but has a declaration of urgent medical need completed by the principal investigator or physician sub-investigator per 21 CFR 1271

Exclusion Criteria

Recipient - Has a suitable HLA-identical sibling or suitable 12/12 (HLA-A, B, C, DRB1, DQB1, and DPB1) HLA-matched unrelated donor available in an appropriate time frame - Any other active malignancy other than the one for which this HCT is indicated - Received a prior allogeneic HCT at any time - Pregnant, if female is of childbearing potential, negative test must be confirmed by serum or urine pregnancy test within 14 days prior to enrollment - If sexually active, agreement to use birth control until 6 months after T cell infusion - Breast feeding - Any severe current uncontrolled bacterial, fungal or viral infection Donor - Pregnant, negative test must be confirmed by serum or urine pregnancy test within 14 days prior to enrollment if female - If female, breast feeding

Study Design

Phase
Phase 1
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
HAPALL Treatment
Patients receive a conditioning regimen that will comprise of ATG, Fludarabine, Cyclophosphamide. Melphalan and Thiotepa. Following the conditioning regimen, patients receive infusion of TCRαβ+/CD19 B cell depleted progenitor cell infusion on day 0. Then as early as day + 14 patients will receive the previously manufactured CD19-CAR(Mem) T cell product. Patients will then be monitored for safety and efficacy of the infused CAR T-cell product, Cells for infusion are prepared using the CliniMACS system.
  • Drug: Anti-Thymocyte Globulin (Rabbit)
    Days -10, -11, -12.
  • Drug: Cyclophosphamide
    60 mg/kg intravenous once daily on day -9.
  • Drug: Fludarabine
    30 mg/m2 intravenous once daily for >10 kg, 1 mg/kg intravenous once daily for ≤10 kg on days -4, -5, -6, -7, -8.
  • Drug: Thiotepa
    5 mg/kg intravenous twice daily on day -3.
  • Drug: Mesna
    Mesna is planned to be administered at 15 mg/kg/dose prior to cyclophosphamide and at approximately 3, 6, and 9 hours after the cyclophosphamide infusion, to give a 1:1 ratio of mesna:cyclophosphamide.
  • Drug: Melphalan
    70 mg/m2 intravenous once daily for >10 kg, 2.3 mg/kg intravenous once daily for ≤10 kg on days -1, and -2.
  • Drug: Filgrastim
    G-CSF* 10 mcg/kg/day SC days 0, -1, -2, -3, -4, -5.
    Other names:
    • G-CSF
  • Device: CliniMACS System
    The mechanism of action of the CliniMACS Cell Selection System is based on magnetic-activated cell sorting (MACS). The CliniMACS device is a powerful tool for the isolation of many cell types from heterogeneous cell mixtures, (e.g. apheresis products). These can then be separated in a magnetic field using an immunomagnetic label specific for the cell type of interest.

Recruiting Locations

St. Jude Children's Research Hospital
Memphis, Tennessee 38105
Contact:
Swati Naik, MBBS
888-226-4343
referralinfo@stjude.org

More Details

Status
Recruiting
Sponsor
St. Jude Children's Research Hospital

Study Contact

Swati Naik, MBBS
888-226-4343
referralinfo@stjude.org

Detailed Description

This is a Phase I study evaluating the addback of CD19-CAR(Mem) T cells after TCRαβ+/CD19 B cell depleted haploidentical donor transplantation for pediatric patients with relapsed/refractory CD19+ B-cell malignancies. Donors that meet eligibility criteria will be consented to undergo two separate collections: 1) G-CSF mobilized stem cell graft via apheresis for progenitor cell infusion and 2) Non-mobilized peripheral blood mononuclear cells (PBMC) via apheresis for subsequent CAR T-cell manufacturing and DLI if needed. Patients that meet eligibility criteria to receive therapy will be consented to proceed on study. Treatment will include a conditioning chemotherapy preparative regimen followed by infusion of TCRαβ/CD19 B cell depleted progenitor cell infusion on day 0. Then as early as day + 14 patients will receive the previously manufactured CD19-CAR(Mem) T cell product. Patients will then be monitored for safety and efficacy of the infused CAR T-cell product, as well as collection of correlative samples.