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)

Lymphoblastic Lymphoma Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05885464
• Other study ID #: BTX-ALO-001
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: May 25, 2023
• Est. completion date: December 2031

Study information

• Verified date: April 2024
• Source: Beam Therapeutics Inc.
• Contact: Medical Information
• Phone: 857-327-8641
• Email: clinicalinfo[@]beamtx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 102
• Est. completion date: December 2031
• Est. primary completion date: December 2031
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 50 Years

Eligibility

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.

Related Conditions & MeSH terms

• Leukemia
• Leukemia, Lymphoid
• Lymphoblastic Leukemia
• Lymphoblastic Lymphoma
• Lymphoma
• Lymphoma, Non-Hodgkin
• Precursor Cell Lymphoblastic Leukemia-Lymphoma
• Precursor T-Cell Lymphoblastic Leukemia-Lymphoma

Intervention

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

Locations

Country	Name	City	State
United States	University of Chicago	Chicago	Illinois
United States	Cleveland Clinic- Taussig Cancer Center	Cleveland	Ohio
United States	Colorado Blood Cancer Institute	Denver	Colorado
United States	The University of Kansas Cancer Center	Fairway	Kansas
United States	Sarah Cannon- TriStar Bone Marrow Transplant	Nashville	Tennessee
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Methodist Hospital - Texas Transplant Institute	San Antonio	Texas
United States	Stanford University School of Medicine	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Beam Therapeutics Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	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
Secondary	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
Secondary	Duration of Response (DOR)		Through study completion, an average of 25 months
Secondary	Relapse-free survival (RFS)		Through study completion, an average of 25 months
Secondary	Overall survival		Through study completion, an average of 25 months
Secondary	Relapse-related mortality		Through study completion, an average of 25 months