Single-arm, prospective, open-label feasibility study evaluating the technical and operational feasibility of manufacturing autologous CD19-directed CAR-T cells (MB-CART19.1) at the point of care for the treatment of relapsed or refractory B-ALL in pediatric and adult patients.
Age ≥ 1 year as long as if deemed fit by treating investigator
CD19 expression must be detected (≥20%) on the malignant cells by flow cytometry.
Patients with relapsed or refractory disease with 0.01% or higher blasts in the bone marrow, or patients with confirmed disease progression as demonstrated by FDG PET-CT or CT/MRI of the affected lymph node or spleen after at least 2 cycles/lines of chemotherapy are eligible for enrollment. For patients with Philadelphia-positive disease, a second-generation or higher TKI must have been utilized in one of the treatment lines.
Patients who have relapsed post alloSCT at least 100 days post-transplant, with no evidence of active graft vs host disease, and no longer taking immunosuppressive agents for at least 30 days prior to enrollment.
Rapidly progressive, uncontrolled disease as assessed by the treating physician and/or principal investigator.
Persistent extramedullary disease.
Isolated CNS and/or testicular disease.
Current autoimmune disease, or history of autoimmune disease with potential CNS involvement
Single group
MB-CART19.1
GeneticAll participants will undergo leukapheresis for collection of autologous T cells, which will then be manufactured into MB-CART19.1 on-site using CliniMACS Prodigy platform. Successfully manufactured MB-CART19.1 products will be infused back to the patient following a lymphodepleting chemotherapy regimen.
Proportion of enrolled patients for whom MB-CART19.1 product is successfully manufactured on-site and meets release criteria.
Assessment of the feasibility and success rate of on-site manufacturing of MB-CART19.1, defined as the proportion of enrolled patients whose cell product is produced and meets established release specifications.
Overall response rate (ORR) (CR, CR with incomplete hematologic recovery (CRh)) on day 28.
Evaluation of overall response rate (ORR) at Day 28, measured as the percentage of patients who achieve complete remission (CR) or complete remission with incomplete hematologic recovery (CRh) following MB-CART19.1 infusion.
Duration of response time from first documented response to progression or death up to 12 months post-infusion
Duration of response time from first documented response to progression or death up to 12 months post-infusion
Rate of measurable residual disease (MRD) negativity at 1-, 3-, 6- and 12-month intervals
Evaluation of rate of measurable residual disease (MRD) negativity at scheduled follow-up visits to monitor clinical status and response post-infusion.
MB-CART19.1 manufacturing turnaround time
Time required to complete on-site manufacturing of MB-CART19.1 from leukapheresis to product release.
Click on the lead sponsor to view all of their trials.