This will be a Phase 1, open-label study to evaluate the safety and efficacy of BEAM-201 in patients with R/R T-ALL or T-LLy. BEAM-201 is an allogeneic anti-CD7 CART therapy.
Despite favorable outcomes in newly diagnosed patients, approximately 20% of pediatric and young adult T-ALL patients and 40% of adult patients will have refractory disease or will relapse within 2 years of their initial diagnosis. Survival rates of patients with relapsed disease remain below 35%. For recurrent disease, allogeneic hematopoietic stem cell transplant (HSCT) is the only known potentially curative treatment. However, a prerequisite to HSCT is obtaining a complete remission, which remains a significant challenge as only 40 to 50% of patients achieve a second remission with current reinduction regimens with salvage rates even lower for patients with disease that is refractory to first-line chemotherapy. BEAM-201 is an allogeneic anti-CD7 CAR T cell product that has shown promising early evidence of efficacy, with 3 out of 4 adult T-ALL patients infused had a CRi/CR ≥28 days after infusion. Safety of BEAM-201 has been favorable and consistent with known adverse events associated with other CAR T cell therapies. Given the current treatment landscape of T-ALL/T-LLy, promising clinical experience with BEAM-201, and that \>98% of T-ALL cases highly and homogenously express CD7 protein on the surfaces of their lymphoblasts, the investigators think there is compelling rationale in further investigating the safety and efficacy of BEAM-201 in pediatric patients.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
33
The investigational agent in this protocol is allogeneic anti-CD7 CART cells (BEAM-201). BEAM-201 is comprised of allogeneic anti-CD7 CAR-T cells edited by 4 gRNAs and a single mRNA encoding a CBE, then transduced with a lentiviral vector (LVV) encoding the anti-CD7 chimeric antigen receptor (CAR) molecule.
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
RECRUITINGDetermine the Maximum Tolerate Dose of Beam 201 Cells
The Maximum Tolerated Dose will be determined by measuring the incidence of dose limiting toxicities following administration of the product.
Time frame: 5 years
Frequency of Adverse Events Following Beam-201 administration
Frequency of Adverse events will be measured by evaluating the frequency and severity of treatment related adverse events following administration of Beam-201 Cells
Time frame: 5 years
• Determine the overall response rate following BEAM-201 infusion
Defined as the frequency of patients who achieve complete remission or partial response following treatment with Beam 201
Time frame: 5 years
Determine depth of response based on MRD for patients with clinical responses following BEAM-201 infusion
Evaluated as the frequency of patients who have minimal residual disease through next generation sequencing
Time frame: 5 years
Determine the proportion of patients treated with BEAM-201 who are deemed appropriate for stem cell transplant
As evaluated the percentage of patients who proceed to stem cell transplant following Beam-201 administration
Time frame: 5 years
• Determine duration of response for patients with clinical responses following BEAM 201 infusion
Duration of Response will be measured by the average number of months in remission after Beam 201 administration
Time frame: 5 years
Determine overall survival following BEAM-201 infusion
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Overall survival will be measured as proportion of subjects who remain alive 12 months after administration with Beam-201 cells
Time frame: 5 years