Phase 1 Study of Niclosamide (ANA001) in Pediatric Patients With Relapsed and Refractory AML

Stanford University1 个研究点分布在 1 个国家目标入组 16 人2022年11月21日

适应症Acute Myeloid Leukemia (AML)

干预措施Niclosamide

概览

阶段: 1 期
干预措施: Niclosamide
疾病 / 适应症: Acute Myeloid Leukemia (AML)
发起方: Stanford University
入组人数: 16
试验地点: 1
主要终点: Dose-limiting toxicity
状态: 招募中
最后更新: 3个月前

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验

概览

简要总结

Protocol is designed to evaluate a niclosamide dose escalation scale in combination with cytarabine as a therapeutic modality for pediatric subjects with relapsed/refractory acute myeloid leukemia.

注册库

clinicaltrials.gov

开始日期

2022年11月21日

结束日期

2026年12月1日

最后更新

3个月前

研究类型

Interventional

研究设计

Sequential

性别

All

研究者

发起方

Stanford University

责任方

Sponsor

入排标准

入选标准

•1\. Prior morphologically-confirmed diagnosis of AML based on WHO Criteria
•Has previously failed all available and suitable therapies for AML. Disease relapse or the presence of refractory disease after ≥ 2 cycles of intensive chemotherapy; or ≥ 4 cycles of non-intensive chemotherapy or hypomethylating agents (HMAs) must be documented by bone marrow (BM) examination demonstrating ≥ 5% blasts in the BM by morphology or ≥ 1% blasts by flow cytometry,
•5% blasts in the peripheral blood (confirmed by flow cytometry, cytogenetics or FISH), ≥ 1% MRD
•\+ by flow cytometry, FISH, PCR or NGS, and not attributable to another cause (EXCEPTION: subjects with frank disease progression in the face of treatment with HMA with or without venetoclax will be considered eligible regardless of treatment cycles administered if they meet the other eligibility criteria). No prior treatment with niclosamide.
•Age ≥ 2 and ≤ 30 years
•Body surface area (BSA) ≤ 2.10 m2
•, calculated per the Mostellar formula
•Must be able to tolerate po or ng medications.
•Performance status: Subject ≤ 16 years old: Lansky ≥ 50 Subject \> 16 years old: Karnofsky ≥ 50%
•Life expectancy of greater than 4 weeks

排除标准

•Received anticancer therapy (chemotherapy, immunotherapy, radiotherapy, or investigational therapy) within 2 weeks prior to starting study treatment. Administration of hydroxyurea 10 to 20 mg/kg/day PO (maximum 1000 mg PO BID) to control high WBC \> 50 x 103
•/mm3 is permitted at MD discretion (however, hydroxyurea should be stopped at least 24 hours prior to protocol therapy start).
•Receiving any other investigational agents, including niclosamide.
•Unresolved toxicities due to prior anticancer therapy, defined as not having resolved to Grade 0 or 1 (by CTCAE version 5 criteria), unless otherwise defined in the inclusion/exclusion criteria with the exception of alopecia
•Acute promyelocytic leukemia (French-American-British Class M3-AML)
•Known active central nervous system (CNS) leukemia; subjects can enroll on study if CNS disease can be cleared with intrathecal chemotherapy, in the judgement of the treating physician
•Known congenital bleeding disorders, including but not limited to hemophilia
•Known active uncontrolled systemic infection
•Malabsorption syndrome, disease significantly affecting gastrointestinal function, or resection of the stomach or small bowel, uncontrolled symptomatic inflammatory bowel disease or ulcerative colitis, or partial or complete bowel obstruction, at the time of study entry
•Inability to receive administration of niclosamide in the available formulation(s)

研究组 & 干预措施

Niclosamide 250 mg/m2 /day divided BID

干预措施: Niclosamide

Niclosamide 500 mg/m2 /day divided BID

干预措施: Niclosamide

Niclosamide 800 mg/m2 /day divided BID

干预措施: Niclosamide

Niclosamide 1200 mg/m2 /day divided BID

干预措施: Niclosamide

结局指标

主要结局

Dose-limiting toxicity

时间窗: 30 days

Dose-limiting toxicities (DLTs) are defined as any events ≥ Grade 3 that are at least possibly, probably, or definitely related to niclosamide treatment