NCT07170644进行中（未招募）2 期
A Single-arm, Open-label Phase 2 Study of Nirogacestat in Adult Japanese Patients With Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF)
SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany4 个研究点 分布在 1 个国家目标入组 20 人开始时间: 2025年8月8日最近更新:
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 20
- 试验地点
- 4
- 主要终点
- Objective response rate (ORR)
研究概览
简要总结
This study is being conducted to characterize the efficacy and safety of nirogacestat in Japanese adults with progressing desmoid tumors/aggressive fibromatosis.
详细描述
Desmoid tumors, also referred to as aggressive fibromatosis, are rare, locally invasive, slow growing soft tissue tumors. Although considered benign because of their inability to metastasize, desmoid tumors can cause significant morbidity and occasionally mortality in patients.
Nirogacestat is a tumor inhibitor that works by slowing or stopping the growth of tumor cells. Nirogacestat is a tablet taken by mouth and has been approved in the USA for adult patients with progressing desmoid tumors who require systemic treatment.
This is an open-label study to characterize the efficacy and safety of nirogacestat in Japanese adults with progressing desmoid tumors/aggressive fibromatosis (DT).
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
- •Participant is aged ≥18 at the time of signing the informed consent.
- •Participant has histologically confirmed DT (by local pathologist prior to informed consent) that has progressed by ≥20% as measured by RECIST v1.1 within 12 months of the screening visit scan.
- •Participant has:
- •Treatment-naive, measurably progressing DT that is deemed not amenable to surgery without the risk of significant morbidity; OR
- •Recurrent, measurably progressing DT following at least 1 line of therapy; OR
- •Refractory, measurably progressing DT following at least 1 line of therapy.
- •Participant agreed to provide archival or new tumor tissue for re-confirmation of disease.
- •Participant has a DT tumor where continued PD will not result in immediate significant risk to the participant.
- •Participants who are receiving chronic NSAIDs as treatment for conditions other than DT must be receiving them prior to documented DT progressive disease (inclusion criterion 2) and on a stable dose for at least 28 days prior to the first dose of study treatment.
- •Participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤2 at screening
- •Participant has adequate organ and bone marrow function
排除标准
- •Participant has known malabsorption syndrome or preexisting gastrointestinal conditions that may impair absorption of nirogacestat.
- •Participant has experienced any of the following within 6 months of signing informed consent: clinically significant cardiac disease (New York Heart Association Class III or IV), myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident, transient ischemic attack, or symptomatic pulmonary embolism.
- •Participant has had lymphoma, leukemia, or any malignancy within the past 5 years at the time of informed consent, except for any locally recurring cancer that has been treated curatively (e.g., resected basal or squamous cell skin cancer, superficial bladder cancer, carcinoma in situ of the cervix or breast), with no evidence of metastatic disease for 3 years at the time of informed consent.
- •Participant has known severe hepatic impairment
- •Participant previously received or is currently receiving gamma secretase inhibitors or anti-Notch antibody therapy
- •Participant is currently using any treatment for DT/AF including tyrosine kinase inhibitors (TKIs) or any investigational treatment 28 days (or 5 half-lives, whichever is longer) prior to the first dose of study treatment
- •Participant is currently using or anticipates using food or drugs that are known strong/moderate cytochrome P450 (CYP) 3A4 inhibitors, or strong CYP3A inducers within 14 days prior to the first dose of study treatment.
- •Participant has experienced other severe acute or chronic medical or psychiatric conditions within 1 year of signing informed consent.
- •Participant is unable to comply with study related procedures (including, but not limited to, the completion of electronic patient-reported outcomes)
研究组 & 干预措施
Nirogacestat
Experimental
Nirogacestat 150 mg by mouth, twice daily
干预措施: Nirogacestat oral tablet (Drug)
结局指标
主要结局
Objective response rate (ORR)
时间窗: On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.
Objective response rate (ORR), defined as the proportion of participants with confirmed complete response (CR) + partial response (PR) assessed by independent Central Imaging Review using RECIST v1.1 (Eisenhauer 2009).
次要结局
- Duration of response (DoR)(On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.)
- Time to Response (TTR)(On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.)
- Safety endpoints will include incidence of treatment-emergent adverse events (TEAEs), changes in laboratory parameters, vital signs, physical examination findings, and ECGs.(On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 5 years.)
- Progression Free Survival (PFS)(On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.)
- Symptoms and impacts will be assessed by evaluating change from baseline at Cycle 10 on the following patient reported outcomes (PROs):(On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.)
- Characterize nirogacestat serum concentrations.(On the first day of the first cycle and of every third cycles (each cycle is 28 days) until disease progression is observed or death, assessed up to approximately 3 years.)
研究者
研究点 (4)
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