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)

• ClinicalTrials.gov Identifier: NCT05885464
• Recruitment Status: Recruiting
• First Posted: June 2, 2023
• Last Update Posted: October 25, 2023
• Sponsor: Beam Therapeutics Inc.
• Information provided by (Responsible Party): Beam Therapeutics Inc.

Tracking Information

• First Submitted Date: May 23, 2023
• First Posted Date: June 2, 2023
• Last Update Posted Date: October 25, 2023
• Actual Study Start Date: May 25, 2023
• Estimated Primary Completion Date: December 2031 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: June 1, 2023)

• 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) [ Time Frame: 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 [ Time Frame: From treatment with BEAM-201 through study completion ]

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: June 1, 2023)

• 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 [ Time Frame: 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 [ Time Frame: Through study completion, an average of 25 months ]
• Duration of Response (DOR) [ Time Frame: Through study completion, an average of 25 months ]
• Relapse-free survival (RFS) [ Time Frame: Through study completion, an average of 25 months ]
• Overall survival [ Time Frame: Through study completion, an average of 25 months ]
• Relapse-related mortality [ Time Frame: Through study completion, an average of 25 months ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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)
• Official Title: A Phase 1/2, Dose-Exploration and Dose-Expansion Study Evaluating the Safety and Efficacy of Multiplex Base-Edited, Allogeneic Anti-CD7 CAR-T Cells (BEAM-201) in Relapsed/Refractory T-Cell Acute Lymphoblastic Leukemia (T-ALL) or T-Cell Lymphoblastic Lymphoma (T-LL)
• 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 Provided
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2

Study Design

Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

The maximum number of patients for this study is approximately 102 patients:

• 36 patients in the Phase 1 dose exploration
• approximately 12 patients in the Phase 1 dose-expansion cohorts
• 6 patients in the pediatric cohort
• approximately 48 patients in the Phase 2 cohort.

Masking: None (Open Label)
Primary Purpose: Treatment

Condition

• Lymphoblastic Lymphoma
• T-Cell Lymphoblastic Leukemia/Lymphoma
• Lymphoblastic Leukemia

Intervention

Biological: BEAM-201

A single dose of BEAM-201 administered by IV following one of two lymphodepletion regimens

Study Arms

• Experimental: Fludarabine, cyclophosphamide and alemtuzumab
  Lymphodepletion regimen including fludarabine, cyclophosphamide and alemtuzumab
  Intervention: Biological: BEAM-201
• Experimental: Fludarabine, cyclophosphamide without alemtuzumab
  Lymphodepletion regimen without Alz but consisting of the same dose of Flu/Cy as in the other arm
  Intervention: Biological: BEAM-201

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: June 1, 2023): 102
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 2031
• Estimated Primary Completion Date: December 2031 (Final data collection date for primary outcome measure)

Eligibility Criteria

Key Inclusion Criteria:

1. Ages 18 to ≤ 50 years.
2. Ages ≥ 1 year to < 18 years, after health authority approval.

3. 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:

   1. 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

   2. 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.
4. 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:

1. CNS involvement meeting any of the following criteria: CNS-3 disease, progressive CNS involvement despite therapy, CNS parenchymal or cranial nerve lesions on imaging.
2. Clinically active CNS dysfunction or known history of irreversible neurological toxicity related to prior antileukemic therapy.
3. Receipt of prior CD7 targeted therapy.
4. Systemic antileukemic therapy intended to induce or maintain remission within 14 days prior to completion of screening.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 50 Years (Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Medical Information; 857-327-8641; clinicalinfo@beamtx.com

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT05885464
• Other Study ID Numbers: BTX-ALO-001
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Beam Therapeutics Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: Beam Therapeutics Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Beam Therapeutics Inc.
• Verification Date: October 2023