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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Fludarabine, cyclophosphamide and alemtuzumab | Experimental | Lymphodepletion regimen including fludarabine, cyclophosphamide and alemtuzumab |
|
| Fludarabine, cyclophosphamide without alemtuzumab | Experimental | Lymphodepletion regimen without Alz but consisting of the same dose of Flu/Cy as in the other arm |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BEAM-201 | Biological | A single dose of BEAM-201 administered by IV following one of two lymphodepletion regimens |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
| Proportion of patients treated with BEAM-201 deemed appropriate for HSCT based on investigator assessment of clinical response |
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Key Inclusion Criteria:
Ages 18 to ≤ 50 years.
Ages ≥ 1 year to < 18 years, after health authority approval.
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:
Second or greater relapse or post-transplant relapse, defined as:
Refractory disease, defined as:
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 Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University School of Medicine | Stanford | California | 94304 | United States | ||
| Colorado Blood Cancer Institute |
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The maximum number of patients for this study is approximately 102 patients:
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| Through study completion, an average of 25 months |
| Duration of Response (DOR) | Through study completion, an average of 25 months |
| Relapse-free survival (RFS) | Through study completion, an average of 25 months |
| Overall survival | Through study completion, an average of 25 months |
| Relapse-related mortality | Through study completion, an average of 25 months |
| Denver |
| Colorado |
| 80218 |
| United States |
| University of Chicago | Chicago | Illinois | 60637 | United States |
| The University of Kansas Cancer Center | Fairway | Kansas | 66205 | United States |
| Dana Farber and Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Cleveland Clinic- Taussig Cancer Center | Cleveland | Ohio | 44106 | United States |
| OHSU Knight Cancer Institute Hematology Oncology | Portland | Oregon | 97239 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Sarah Cannon- TriStar Bone Marrow Transplant | Nashville | Tennessee | 37203 | United States |
| Methodist Hospital - Texas Transplant Institute | San Antonio | Texas | 78229 | United States |
| ID | Term |
|---|---|
| D054198 | Precursor Cell Lymphoblastic Leukemia-Lymphoma |
| D008223 | Lymphoma |
| ID | Term |
|---|---|
| D007945 | Leukemia, Lymphoid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
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