临床试验/2025-521671-31-00

2025-521671-31-00招募中2 期

A Phase 1b/2 Study to Investigate the Safety, Efficacy and Pharmacokinetics of Administration of Subcutaneous (SC) Blinatumomab in Pediatric Participants with Relapsed/Refractory (R/R) and Minimal Residual Disease Positive (MRD+) B Cell Precursor Acute Lymphoblastic Leukemia (B-ALL)

Amgen Inc.9 个研究点分布在 2 个国家目标入组 5 人开始时间: 2026年2月13日最近更新: 2026年2月13日

试验速览

阶段: 2 期
状态: 招募中
发起方
入组人数: 5
试验地点: 9
主要终点: Phase 1b: Dose limiting toxicities (DLTs), treatment-emergent adverse events (TEAE), serious TEAE, treatment related TEAE, and adverse events of interest (EOI)

研究概览研究设计入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

Phase 1b: To evaluate the safety and tolerability of SC blinatumomab in children below 12 years of age Phase 2 Cohort Ph2-R & M: To evaluate the efficacy of SC blinatumomab

研究设计

分配方式: Not Applicable
主要目的: Phase 2
盲法: None

入排标准

年龄范围: 0 years 至 17 years（0-17 Years）
接受健康志愿者: 是

入选标准

•Participant’s legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent, and the participant has provided written assent based on local regulations and/or guidelines before any study-specific activities/procedures being initiated.
•Age 28 days to < 12 years at the time of consent
•Lansky Performance Status (LPS) of ≥ 50%
•Prior CD19-directed therapy such as blinatumomab or CD19 chimeric antigen receptor T cells (CAR-T) cells will be allowed (with demonstrated continued CD19+ expression) if treatment ended >4 weeks prior to start of protocol therapy and no prior central nervous system (CNS) complications (see CNS exclusion criteria below).
•Any Philadelphia chromosome-positive (Ph+) participant intolerant or refractory to prior tyrosine kinase inhibitors (TKIs) are eligible.
•For Phase 1b and Phase 2 cohort in participants with R/R B-ALL (Ph2-R): Participants with B-ALL relapsed after or refractory to any line of treatment including allogeneic HSCT  Refractory disease is defined as the failure to achieve CR.  Relapsed disease is defined as isolated hematological relapse or combined hematological and extramedullary (EM) relapse after achieving a CR. Greater than or equal to 5% blasts in the BM is considered as relapse in the BM.
•For Phase 2 cohort in participants with MRD+ B-ALL (Ph2-M): Participants with MRD+ B-ALL must have between ≥ 0.1% and < 5% blasts in the BM. With a peripheral blood count as defined below: ANC (neutrophils) ≥ 500/μL; Platelets ≥ 50,000/μL (transfusion permitted)
•For Phase 2 cohort in participants with MRD+ B-ALL (Ph2-M):Submission of an archival bone marrow specimen from primary diagnosis or relapse for clone-specific calibration of MRD. See section 8.2.9 and the lab manual for minimum sample requirements. For patients in the US and Canada that have a prior Adaptive clonoSEQ result from bone marrow testing that can be made available for use in this study for clone specific calibration, this may be acceptable in lieu of the archival bone marrow specimen with approval by the Medical Monitor. The participant must also provide consent to the use of the prior clonoSEQ result.

排除标准

•Disease Related: Active ALL in the CNS. If CSF leukemia is present, participants must receive intrathecal therapy and have documented negative CSF prior to enrolling.
•Prior/Concomitant Therapy: Symptoms and/or clinical signs and/or radiological and/or sonographic signs that indicate an acute or uncontrolled chronic infection, any other concurrent disease or medical condition that could be exacerbated by the treatment or would seriously complicate compliance with the protocol
•Prior/Concomitant Therapy: Allogeneic HSCT within 12 weeks before the start of blinatumomab
•Prior/Concomitant Therapy: Cancer chemotherapy within 2 weeks before the start of protocol specified therapy, with the exception of intrathecal chemotherapy and/or low dose maintenance therapy (for example vinca alkaloids, mercaptopurine, methotrexate, or hydroxyurea). Any low dose chemotherapy as stated above must be discontinued before starting pre-phase chemotherapy and/or dexamethasone.
•Prior/Concomitant Therapy: Immunotherapy (eg, rituximab, alemtuzumab) within 4 weeks before start of protocol specified therapy
•Prior/Concurrent Clinical Study Experience: Currently receiving treatment in another investigational device or drug study, or less than 30 days or 5 half-lives since ending treatment in another investigational device or drug study(ies). This does not apply to other investigational procedures or participation in observational research studies.
•Diagnostic Assessments: Abnormal screening laboratory values as defined below: Direct bilirubin > 3.0 mg/dL prior to start of treatment; Estimated Glomerular Filtration Rate (eGFR) < 30 mL/min. per 1.73 m2 per the Revised Schwartz equation for children 2 to < 12 years. For children < 2 years of age, at least 50% of age and gender appropriate normal
•Other Exclusions: Participant likely to not be available to complete all protocol-required study visits or procedures, and/or to comply with all required study procedures (eg, Clinical Outcome Assessments) to the best of the Participant, Participant’s legal guardian’s, and investigator’s knowledge. Completion of patient reported outcomes questionnaires is not required and will not be prohibitive to enrollment in case patient has intellectual disability or cognitive impairment or in case instrument is unavailable in subject’s language.
•History or evidence of any other clinically significant disorder, condition, or disease (except for those outlined above) that, in the opinion of the investigator or Amgen physician, if consulted, would pose a risk to participant safety, or interfere with the study evaluation, procedures or completion. Completion of patient reported outcomes questionnaires is not required and will not be prohibitive to enrollment in case patient has intellectual disability or cognitive impairment or in case instrument is unavailable in subject’s language.
•Other Medical Conditions: History of other malignancy within the past 3 years, with the following exception: Malignancy treated with curative intent and with no known active disease present for ≥ 3 years before enrollment and felt to be at low risk for recurrence by the treating physician.
•Other Medical Conditions: History or presence of clinically relevant CNS pathology or event such as epilepsy, childhood seizure, paresis, aphasia, stroke, severe brain injuries, cerebellar disease, organic brain syndrome, psychosis, or severe (≥ grade 3) CNS events including ICANS from prior CAR-T or other T-cell engager therapies.
•Other Medical Conditions: Isolated EMdisease
•Other Medical Conditions: Current autoimmune disease or history of autoimmune disease with potential CNS involvement.
•Other Medical Conditions: Patients with Down Syndrome are not eligible for this study
•Prior/Concomitant Therapy: Known hypersensitivity to blinatumomab or to any component of the product formulation. Sensitivity to any of the products administered during dosing.
•Prior/Concomitant Therapy: Active acute or chronic graft versus host disease requiring systemic treatment with immunosuppressive medication.
•Prior/Concomitant Therapy: Known infection with human immunodeficiency virus (HIV) or chronic infection with hepatitis B virus or hepatitis C virus.

结局指标

主要结局

Phase 1b: Dose limiting toxicities (DLTs), treatment-emergent adverse events (TEAE), serious TEAE, treatment related TEAE, and adverse events of interest (EOI)

Phase 2 R/R B-ALL (Cohort Ph2-R): CR/CRh within the first 2 cycles

Phase 2 MRD+ B-ALL (Cohort Ph2-M): CR with MRD negative response (MRD <10-4 [0.01%]) within the first 2 cycles

次要结局

Phase 1b: Complete remission/complete remission with partial hematological recovery (CR/CRh) within the first 2 cycles
Phase 1b: CR within the first 2 cycles
Phase 1b: CR/CRh/CRi/Blast free hypoplastic or aplastic BM within the first 2 cycles
Phase 1b: MRD negative response (MRD level < 10-4 [0.01%]) within the first 2 cycles
Phase 1b: DOR is defined as the time from the first response of CR/CRh within the first 2 cycles until hematological relapse (Including EM relapse) per investigator’s assessment or death due to any cause, whichever occurs first
Phase 1b: PK parameters following SC blinatumomab administration including, but not limited to, maximum concentration (Cmax), time to maximum concentration (Tmax), and area under the concentration time curve (AUC)
Phase 1b: Incidence of anti-blinatumomab antibody formation
Phase 2 (Cohort Ph2-R) Key Secondary: CR/CRh with MRD negative response (MRD < 10-4 [0.01%]) within the first 2 cycles
Phase 2 (Cohort Ph2-R) Key Secondary: DOR is defined as the time from the first response of CR/CRh within the first 2 cycles until hematological relapse (Including EM relapse) per investigator’s assessment or death due to any cause, whichever occurs first
Phase 2 (Cohort Ph2-R): CR within the first 2 cycles
Phase 2 (Cohort Ph2-R): CR/CRh/CRi/Blast free hypoplastic or aplastic BM within the first 2 cycles
Phase 2 (Cohort Ph2-R): OS is defined as the time from the first dose until death due to any cause
Phase 2 (Cohort Ph2-R): Incidence of TEAE, serious TEAE, Treatment related TEAE, and EOI
Phase 2 (Cohort Ph2-R): Blinatumomab PK serum concentrations
Phase 2 (Cohort Ph2-R): Incidence of anti-blinatumomab antibody formation
Phase 2 (Cohort Ph2-M) Key Secondary: CR/CRh with MRD negative response (MRD < 10-4 [0.01%]) within the first 2 cycles
Phase 2 (Cohort Ph2-M) Key Secondary: DOR is defined as the time from the first response CR with MRD negative response (MRD < 10-4 [0.01%]) within the first 2 cycles until hematological relapse (including EM relapse) or MRD relapse (MRD ≥ 10-4 [0.01%]) per investigator’s assessment or death due to any cause, whichever occurs first
Phase 2 (Cohort Ph2-M): CR/CRh/CRi/Blast free hypoplastic or aplastic BM with MRD negative response (MRD < 10-4 [0.01%]) within the first 2 cycles
Phase 2 (Cohort Ph2-M): OS, defined as the time of the start of first dose of SC blinatumomab until death due to any cause
Phase 2 (Cohort Ph2-M): Incidence of TEAE, serious TEAE, Treatment related TEAE, and EOI
Phase 2 (Cohort Ph2-M): Blinatumomab PK serum concentrations
Phase 2 (Cohort Ph2-M): Incidence of anti-blinatumomab antibody formation

研究者

发起方

Amgen Inc.

申办方类型

Pharmaceutical company

责任方

Principal Investigator

主要研究者

Medical Information

Scientific

Amgen Inc.

研究点 (9)

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