NCT05885464进行中（未招募）1 期
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)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 5
- 试验地点
- 10
- 主要终点
- 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
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 50 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Fludarabine, cyclophosphamide and alemtuzumab
Experimental
Lymphodepletion regimen including fludarabine, cyclophosphamide and alemtuzumab
干预措施: BEAM-201 (Biological)
Fludarabine, cyclophosphamide without alemtuzumab
Experimental
Lymphodepletion regimen without Alz but consisting of the same dose of Flu/Cy as in the other arm
干预措施: BEAM-201 (Biological)
结局指标
主要结局
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
时间窗: From treatment with BEAM-201 through study completion
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)
时间窗: Through study completion, an average of 25 months
次要结局
- 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)
- Overall survival(Through study completion, an average of 25 months)
- Duration of Response (DOR)(Through study completion, an average of 25 months)
- Relapse-related mortality(Through study completion, an average of 25 months)
- 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)
- Relapse-free survival (RFS)(Through study completion, an average of 25 months)
研究者
研究点 (10)
Loading locations...
相似试验
已完成
1 期
A Study to Evaluate Immunotherapy Combinations in Participants With Lung CancerNon Small Cell Lung Cancer MetastaticNon Small Cell Lung CancerNonsquamous Nonsmall Cell Neoplasm of LungSensitizing EGFR Gene MutationNCT03846310Arcus Biosciences, Inc.77
终止
1 期
Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Preliminary Clinical Activity of Defactinib in Combination With Avelumab in Epithelial Ovarian CancerEpithelial Ovarian CancerNCT02943317Verastem, Inc.98
已完成
1 期
A Study to Evaluate AB308 in Combination With AB122 in Participants With Advanced MalignanciesAdvanced Solid TumorNon Small Cell Lung Cancer (NSCLC)Lymphoma, Non-HodgkinDiffuse Large B Cell Lymphoma (DLBCL)Gastroesophageal Junction AdenocarcinomaMultiple MyelomaGastric CancerMelanomaCervical CancerEsophageal CancerNCT04772989Arcus Biosciences, Inc.94
招募中
1 期
A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)Glycogen Storage Disease Type IaNCT06735755Beam Therapeutics Inc.36
招募中
1 期
Safety and Efficacy Study of NGGT002 in Adult Patients With PhenylketonuriaPhenylketonuriasNCT06687733NGGT (Suzhou) Biotechnology Co., Ltd.18