NCT04109482终止1 期
A Phase 1/2, Open Label, Multicenter Trial to Assess the Safety and Efficacy of MB-102 in Patients With Relapsed or Refractory Blastic Plasmacytoid Dendritic Cell Neoplasm
Mustang Bio4 个研究点 分布在 1 个国家目标入组 3 人开始时间: 2020年2月17日最近更新:
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 3
- 试验地点
- 4
- 主要终点
- Phase 1: Safety and Tolerability as measured by the number of patients with treatment related adverse events
研究概览
简要总结
A phase 1/2 study to assess the safety and efficacy of MB-102 in patients with relapsed or refractory BPDCN
详细描述
The Phase 1 portion of the study will determine the maximum tolerated dose of MB-102.
The Phase 2 portion of the trial will evaluate the efficacy of MB-102 in relapsed or refractory BPDCN.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Blastic Plasmacytoid Dendritic Cell Neoplasm
- •Patients with a diagnosis of BPDCN according to WHO classification (Arber et al., 2016) confirmed by hematopathology and histological/cytological evidence of BPDCN in the peripheral blood, bone marrow, spleen, lymph nodes, skin and/or other sites who have failed one prior therapy.
- •General Inclusion Criteria
- •Male and female patients ≥ 18 years of age at the time of consent.
- •Written informed consent in accordance with federal, local, and institutional guidelines.
- •Must be able to adhere to the study visit schedule and other protocol requirements.
- •Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or
- •Meet the following laboratory criteria:
- •Absolute lymphocyte count (ALC) > 100/mm3
- •ALT/SGPT and AST/SGOT < 2.5x the upper limit of normal (ULN) unless due to underlying disease state
- •Calculated creatinine clearance ≥ 45.0 mL/min as estimated by Cockcroft Gault and dialysis independent
- •Total bilirubin ≤ 3.0 mg/dL
- •Patients with Gilbert's Syndrome must have a total bilirubin < 5.0 mg/dL.
- •Serum albumin ≥ 3.2 g/dL
- •Cardiac ejection fraction ≥ 45%, with no evidence of pericardial effusion as determined by an echocardiogram (ECHO) or if not available, a multigated acquisition scan (MUGA).
- •Females participants of childbearing potential must have a negative serum test.
- •Patients must agree to use a highly effective method of contraception if procreative potential exists from the start of the study until one year after the completion of lymphodepletion for females and 4 months after completion of lymphodepletion for males.
- •Patients with a previously treated malignancy if treatment of that malignancy was completed greater than 2 years before screening and the patient has no evidence of disease at the time of screening.
- •Patients who have previously undergone allogenic or autologous bone marrow transplants are allowed.
- •Centrally confirmed CD-123 positivity on the bone marrow, or for patients without bone marrow involvement local pathology assessments within 28 days from Screening, showing evidence of CD-123 positivity of skin/lymph node biopsy.
排除标准
- •Patients with a corticosteroid dependence on doses greater than physiological replacement i.e., prednisone no more than 7.5 mg/day or hydrocortisone less than 12mg/m2/day.
- •Contraindication or hypersensitivity to fludarabine or cyclophosphamide.
- •Hypersensitivity or known history of allergic reactions attributed to tocilizumab, Cetuximab, or other anti-EGFR -monoclonal antibodies.
- •Immunotherapy treatments within 28 days prior to leukapheresis.
- •Previous treatment with anti-CD123 CAR-T treatment.
- •Previous treatment with non-CAR-T anti-CD123 agents is allowed e.g. tagraxofusp-erzs.
- •Previous treatment with any other antileukemic or investigational agent within 7 days of leukapheresis.
- •Hydroxyurea is allowed up to 3 days prior to leukapheresis.
- •Patients with history or active seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease or any autoimmune disease with CNS involvement.
- •Patients with known CNS leukemic involvement that are refractory to intrathecal chemotherapy and/or cranio-spinal radiation that have NOT been effectively treated to complete remission (defined as < 5 WBC/mm3 and no blasts in CSF).
- •Patients with active Graft versus Host Disease (GVHD).
- •Acute active infection
- •Patients being administered prophylactic antibiotics, antivirals, or antifungals are permitted.
- •Patients who have any form of primary immunodeficiency, such as severe combined immunodeficiency disease, human immunodeficiency virus (HIV), or acquired immune deficiency syndrome (AIDS).
- •Active infection with hepatitis B or C.
- •Patients requiring supplemental oxygen or mechanical ventilation or oxygen saturation < 92% on room air.
- •Patients with an oxygen saturation < 92%, a pulmonary function test with a result of Diffusing capacity of the lungs for carbon monoxide (DLCO) of ≥ 40% of predicted and a forced expiratory volume in one second (FEV1) > 45% predicted will be accepted.
- •Patients with decompensated hepatic cirrhosis/liver failure.
- •Pregnant or lactating females.
- •Any other clinically significant medical disease or condition that, in the investigator's opinion, may interfere with protocol adherence or a patient's ability to give informed consent.
研究组 & 干预措施
Relapsed or Refractory BPDCN
Experimental
Treatment with MB-102.
干预措施: MB-102 (Biological)
Relapsed or Refractory BPDCN
Experimental
Treatment with MB-102.
干预措施: Fludarabine (Drug)
Relapsed or Refractory BPDCN
Experimental
Treatment with MB-102.
干预措施: Cyclophosphamide (Drug)
结局指标
主要结局
Phase 1: Safety and Tolerability as measured by the number of patients with treatment related adverse events
时间窗: 28 Days
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 in Phase 1
Phase 1: Maximum Tolerated Dose (MTD) and recommended Phase 2 dose
时间窗: 28 Days
To determine the maximum tolerated dose (MTD) and the recommended Phase 2 dose of MB-102
Phase 2: Response Rate of patients with BPDCN
时间窗: up to 3 years
Relapsed or refractory Blastic Plasmacytoid Dendritic Cell Neoplasm is measured by a response rate which consists of Complete Response and clinical Complete Response and Complete Response with incomplete hematologic recovery (CR + CRc + CRi) at day 28 post infusion
次要结局
- Phase 2: BPDCN - DOR(up to 3 years)
- Phase 2: BPDCN - PFS(up to 3 years)
- Phase 2 - Number of patients showing evidence of replication competent lentivirus(up to 3 years)
- Phase 2: BPDCN - OS(up to 3 years)
- Phase 2: BPDCN - MRD(up to 3 years)
- Phase 2 - Adverse events(up to 3 years)
- Phase 2 -Change from Baseline in the European Organization for Research and Treatment (EORTC) QLQ-C 30 Version 3.0.(up to 3 years)
- Phase 2 - Change from Baseline in the Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT) Version 4.0.(up to 3 years)
研究者
研究点 (4)
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