History of Changes for Study: NCT05885464

Version	Submitted Date	Changes
1	June 1, 2023	None (earliest Version on record)
2	June 2, 2023	Recruitment Status, Study Status and Contacts/Locations
3	July 25, 2023	Study Status
4	August 10, 2023	Study Status
5	August 15, 2023	Contacts/Locations and Study Status
6	September 19, 2023	Study Status and Contacts/Locations
7	October 24, 2023	Study Status and Contacts/Locations
8	January 11, 2024	Contacts/Locations and Study Status
9	April 22, 2024	Study Status and Contacts/Locations

Unique Protocol ID:

BTX-ALO-001

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)

Secondary IDs:

Record Verification:

August 2023

September 2023

Overall Status:

Recruiting

Study Start:

May 25, 2023

Primary Completion:

December 2031 [Anticipated]

Study Completion:

December 2031 [Anticipated]

First Submitted:

May 23, 2023

First Submitted that
Met QC Criteria:

June 1, 2023

First Posted:

June 2, 2023 [Actual]

Last Update Submitted that
Met QC Criteria:

August 15, 2023

September 19, 2023

Last Update Posted:

August 16, 2023 [Actual]

September 21, 2023 [Actual]

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:

Study Type:

Interventional

Primary Purpose:

Treatment

Study Phase:

Phase 1/Phase 2

Interventional Study Model:

Parallel Assignment

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.

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.

Number of Arms:

Masking:

None (Open Label)

Allocation:

Non-Randomized

Enrollment:

102 [Anticipated]

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)
[ 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 ]

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
[ 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 ]

Eligibility


Minimum Age:	18 Years	18 Years
Maximum Age:	50 Years	50 Years
Sex:	All	All
Gender Based:
Accepts Healthy Volunteers:	No	No
Criteria:	Key Inclusion Criteria: 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. Key Exclusion Criteria: 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.	Key Inclusion Criteria: 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. Key Exclusion Criteria: 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.

Central Contact Person:

Medical Information
Telephone: 857-327-8641
Email: clinicalinfo@beamtx.com

Locations:

United States, California

Stanford University School of Medicine
[Recruiting]
Stanford, California, United States, 94304

United States, Colorado

Colorado Blood Cancer Institute - SCRI - PPDS
[Recruiting]
Denver, Colorado, United States, 80218

United States, Ohio

Cleveland Clinic- Taussig Cancer Center
[Recruiting]
Cleveland, Ohio, United States, 44106

United States, Pennsylvania

Children's Hospital of Philadelphia
[Recruiting]
Philadelphia, Pennsylvania, United States, 19104