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)
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1/Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Fludarabine, cyclophosphamide and alemtuzumab Lymphodepletion regimen including fludarabine, cyclophosphamide and alemtuzumab |
Biological: BEAM-201
A single dose of BEAM-201 administered by IV following one of two lymphodepletion regimens
|
| Experimental: Fludarabine, cyclophosphamide without alemtuzumab Lymphodepletion regimen without Alz but consisting of the same dose of Flu/Cy as in the other arm |
Biological: BEAM-201
A single dose of BEAM-201 administered by IV following one of two lymphodepletion regimens
|
Outcome Measures
Primary Outcome Measures
- 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]
Secondary Outcome Measures
- 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 [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]
Eligibility Criteria
Criteria
Key Inclusion Criteria:
-
Ages 18 to ≤ 50 years.
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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:
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Second or greater relapse or post-transplant relapse, defined as:
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BM with ≥ 5% lymphoblasts by morphologic assessment or evidence of extramedullary disease at screening after second documented CR; OR
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Flow cytometric confirmation of relapsed T-ALL of at least 0.1% after second CR documented to have been MRD negative < 0.1%; OR
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Any detectable relapsed disease post-allogeneic HSCT with flow cytometric confirmation of T-ALL of at least 0.1%; OR
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Biopsy confirmed evidence of relapsed T-LL on lymph node biopsy after second CR; OR
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Any detectable disease post-allogeneic transplant with biopsy confirmed evidence of T-LL on lymph node biopsy
- Refractory disease, defined as:
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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
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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.
- 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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CNS involvement meeting any of the following criteria: CNS-3 disease, progressive CNS involvement despite therapy, CNS parenchymal or cranial nerve lesions on imaging.
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Clinically active CNS dysfunction or known history of irreversible neurological toxicity related to prior antileukemic therapy.
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Receipt of prior CD7 targeted therapy.
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Systemic antileukemic therapy intended to induce or maintain remission within 14 days prior to completion of screening.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | United States | 19104 |
Sponsors and Collaborators
- Beam Therapeutics Inc.
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- BTX-ALO-001