A Study Evaluating the Safety and Efficacy of BEAM-201 in Relapsed/Refractory T-Cell Acute Lymphoblastic Leukemia (T-ALL) or T-Cell Lymphoblastic Lymphoma (T-LL)
- Conditions
- Lymphoblastic LeukemiaLymphoblastic LymphomaT-Cell Lymphoblastic Leukemia/Lymphoma
- Interventions
- Biological: BEAM-201
- Registration Number
- NCT05885464
- Lead Sponsor
- Beam Therapeutics Inc.
- Brief Summary
This is a Phase 1/2, multicenter, open-label study to evaluate the safety and efficacy of BEAM-201 in patients with relapsed/refractory T-ALL or T-LL. This study consists of Phase 1 dose-exploration cohorts, Phase 1 dose-expansion cohort(s), a Phase 1 pediatric cohort (will enroll patients ages 1 to \< 12 years), and a Phase 2 cohort.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 102
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Ages 18 to ≤ 50 years.
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Ages ≥ 1 year to < 18 years, after health authority approval.
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T-ALL/T-LL that is CD7-positive (defined as at least 20% of blasts positive for CD7 by flow cytometry or immunohistochemistry based on assessment of the study site's CLIA [Clinical Laboratory Improvement Amendments of 1988] certified facility) in second or greater relapse, first relapse post-transplant relapse, or chemotherapy-refractory disease. Specifically:
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Second or greater relapse or post-transplant relapse, defined as:
- BM with ≥ 5% lymphoblasts by morphologic assessment or evidence of extramedullary disease at screening after second documented CR; OR
- Flow cytometric confirmation of relapsed T-ALL of at least 0.1% after second CR documented to have been MRD negative < 0.1%; OR
- Any detectable relapsed disease post-allogeneic HSCT with flow cytometric confirmation of T-ALL of at least 0.1%; OR
- Biopsy confirmed evidence of relapsed T-LL on lymph node biopsy after second CR; OR
- Any detectable disease post-allogeneic transplant with biopsy confirmed evidence of T-LL on lymph node biopsy
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Refractory disease, defined as:
- Primary refractory T-ALL or T-LL, defined as failure to achieve CR after induction chemotherapy, per investigator assessment and based on biopsy-confirmed evidence of residual T-ALL or T-LL; OR
- Relapsed, refractory disease, defined as > 5% BM blasts or biopsy-confirmed evidence of residual TLL after 1 course of re-induction chemotherapy for patients who have relapsed after previously achieving a CR NOTE: Patients with mixed phenotype acute leukemia with T-cell dominant phenotype may be enrolled if the aforementioned criteria are met.
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Eligible for myeloablative conditioning for and allogeneic HSCT based on the investigator's assessment with an available donor identified by a FACT accredited transplant center.
Key
- CNS involvement meeting any of the following criteria: CNS-3 disease, progressive CNS involvement despite therapy, CNS parenchymal or cranial nerve lesions on imaging.
- Clinically active CNS dysfunction or known history of irreversible neurological toxicity related to prior antileukemic therapy.
- Receipt of prior CD7 targeted therapy.
- Systemic antileukemic therapy intended to induce or maintain remission within 14 days prior to completion of screening.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Fludarabine, cyclophosphamide and alemtuzumab BEAM-201 Lymphodepletion regimen including fludarabine, cyclophosphamide and alemtuzumab Fludarabine, cyclophosphamide without alemtuzumab BEAM-201 Lymphodepletion regimen without Alz but consisting of the same dose of Flu/Cy as in the other arm
- Primary Outcome Measures
Name Time Method Overall response rate as defined as proportion of T-ALL patients achieving complete response (CR) or complete response with incomplete hematologic recovery (CRi) or T-LL patients achieving CR or PR at any point after BEAM-201 infusion From treatment with BEAM-201 through study completion Incidence and severity of treatment-emergent adverse events (TEAEs) and treatment-related adverse events, including serious adverse events (SAEs) and dose-limiting toxicities (DLTs; in Phase 1 only) Through study completion, an average of 25 months
- Secondary Outcome Measures
Name Time Method Overall survival Through study completion, an average of 25 months Proportion of patients treated with BEAM-201 deemed appropriate for HSCT based on investigator assessment of clinical response Through study completion, an average of 25 months Duration of Response (DOR) Through study completion, an average of 25 months Relapse-related mortality Through study completion, an average of 25 months Proportion of patients who achieve MRD negative response (defined as < 0.1%) by flow cytometry or next generation sequencing (NGS) in patients achieving morphologic response Starting at Day 28 and multiple time points up to Month 24 Relapse-free survival (RFS) Through study completion, an average of 25 months
Trial Locations
- Locations (10)
Stanford University School of Medicine
🇺🇸Stanford, California, United States
Colorado Blood Cancer Institute
🇺🇸Denver, Colorado, United States
University of Chicago
🇺🇸Chicago, Illinois, United States
The University of Kansas Cancer Center
🇺🇸Fairway, Kansas, United States
Dana Farber and Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Cleveland Clinic- Taussig Cancer Center
🇺🇸Cleveland, Ohio, United States
OHSU Knight Cancer Institute Hematology Oncology
🇺🇸Portland, Oregon, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Sarah Cannon- TriStar Bone Marrow Transplant
🇺🇸Nashville, Tennessee, United States
Methodist Hospital - Texas Transplant Institute
🇺🇸San Antonio, Texas, United States