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)

NCT05885464 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: BTX-ALO-001
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 10

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.

Conditions

Lymphoblastic Lymphoma; T-Cell Lymphoblastic Leukemia/Lymphoma; Lymphoblastic Leukemia

Keywords

Lymphoblastic Leukemia; Lymphoblastic Lymphoma; Base editing; CAR-T

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (6)

• 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

Study Arms (2)

Fludarabine, cyclophosphamide and alemtuzumab

EXPERIMENTAL

Lymphodepletion regimen including fludarabine, cyclophosphamide and alemtuzumab

Biological: BEAM-201

Fludarabine, cyclophosphamide without alemtuzumab

EXPERIMENTAL

Lymphodepletion regimen without Alz but consisting of the same dose of Flu/Cy as in the other arm

Biological: BEAM-201

Interventions

BEAM-201 BIOLOGICAL

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

Fludarabine, cyclophosphamide and alemtuzumab; Fludarabine, cyclophosphamide without alemtuzumab

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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:
• 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
• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Beam Therapeutics Inc.: lead

Study Sites (10)

Stanford University School of Medicine

Stanford, California, 94304, United States

Location

Colorado Blood Cancer Institute

Denver, Colorado, 80218, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

The University of Kansas Cancer Center

Fairway, Kansas, 66205, United States

Location

Dana Farber and Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Cleveland Clinic- Taussig Cancer Center

Cleveland, Ohio, 44106, United States

Location

OHSU Knight Cancer Institute Hematology Oncology

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Sarah Cannon- TriStar Bone Marrow Transplant

Nashville, Tennessee, 37203, United States

Location

Methodist Hospital - Texas Transplant Institute

San Antonio, Texas, 78229, United States

Location

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma; Lymphoma

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: May 23, 2023
• First Posted: June 2, 2023
• Study Start: May 25, 2023
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: January 13, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (10)