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)

April 22, 2024 updated by: Beam Therapeutics Inc.

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)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

102

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • California
      • Stanford, California, United States, 94304
        • Recruiting
        • Stanford University School of Medicine
    • Colorado
      • Denver, Colorado, United States, 80218
        • Recruiting
        • Colorado Blood Cancer Institute
    • Illinois
      • Chicago, Illinois, United States, 60637
        • Recruiting
        • University of Chicago
    • Kansas
      • Fairway, Kansas, United States, 66205
        • Recruiting
        • The University of Kansas Cancer Center
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Recruiting
        • Cleveland Clinic- Taussig Cancer Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
    • Tennessee
      • Nashville, Tennessee, United States, 37203
        • Recruiting
        • Sarah Cannon- TriStar Bone Marrow Transplant
    • Texas
      • San Antonio, Texas, United States, 78229
        • Recruiting
        • Methodist Hospital - Texas Transplant Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
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)
Time Frame: Through study completion, an average of 25 months
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
From treatment with BEAM-201 through study completion

Secondary Outcome Measures

Outcome Measure
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
Time Frame: Starting at Day 28 and multiple time points up to Month 24
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
Through study completion, an average of 25 months
Duration of Response (DOR)
Time Frame: Through study completion, an average of 25 months
Through study completion, an average of 25 months
Relapse-free survival (RFS)
Time Frame: Through study completion, an average of 25 months
Through study completion, an average of 25 months
Overall survival
Time Frame: Through study completion, an average of 25 months
Through study completion, an average of 25 months
Relapse-related mortality
Time Frame: Through study completion, an average of 25 months
Through study completion, an average of 25 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Beam Therapeutics Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 25, 2023

Primary Completion (Estimated)

December 1, 2031

Study Completion (Estimated)

December 1, 2031

Study Registration Dates

First Submitted

May 23, 2023

First Submitted That Met QC Criteria

June 1, 2023

First Posted (Actual)

June 2, 2023

Study Record Updates

Last Update Posted (Actual)

April 23, 2024

Last Update Submitted That Met QC Criteria

April 22, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BTX-ALO-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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