An Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 50
- 试验地点
- 13
- 主要终点
- Objective Response Rate (ORR) in VHL Disease-Associated RCC Tumors
研究概览
简要总结
This study is designed to investigate belzutifan as a treatment for VHL disease associated RCC.
详细描述
This open-label Phase 2 study will evaluate the efficacy and safety of belzutifan in participants with VHL disease who have at least 1 measurable RCC tumor. Belzutifan will be administered orally and treatment will be continuous. Participants will be evaluated radiologically approximately 12 weeks after initiation of treatment and every 12 weeks thereafter while continuing in the study for a minimum of 3 years and then every 24 weeks or more frequently if clinically indicated. Changes in VHL disease-associated non-RCC tumors will also be evaluated.
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
- •Has a diagnosis of von Hippel Lindau disease, based on a germline VHL alteration
- •Has at least 1 measurable solid RCC tumor and no RCC tumor that requires immediate surgical intervention. The diagnosis of RCC can be radiologic (histologic diagnosis not required). Participants may have VHL disease-associated tumors in other organ systems
排除标准
- •Has received prior treatment with belzutifan or another HIF-2α inhibitor
- •Has had any systemic anti-cancer therapy (includes anti-vascular endothelial growth factor [VEGF] therapy or any systemic investigational anti-cancer agent)
- •Has an immediate need for surgical intervention for tumor treatment
- •Has evidence of metastatic disease on screening imaging
研究组 & 干预措施
Open Label Belzutifan
Participants receive 120 mg belzutifan orally once daily. Participants may continue to receive belzutifan in the absence of unacceptable treatment related toxicity or unequivocal disease progression.
干预措施: Belzutifan (Drug)
结局指标
主要结局
Objective Response Rate (ORR) in VHL Disease-Associated RCC Tumors
时间窗: Up to approximately 4 years
ORR is defined as the percentage of participants in the analysis population who have a best confirmed response of Complete Response (CR: Disappearance of all target lesions) or a Partial Response (PR: At least a 30% decrease in the sum of diameters of target lesions) per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST 1.1). ORR will be assessed by independent review committee (ICR) for the primary analysis.
次要结局
- Duration of Response (DOR) in VHL Disease-Associated RCC Tumors(Up to approximately 4 years)
- Time to Response (TTR) in VHL Disease-Associated RCC Tumors(Up to approximately 4 years)
- Progression-free Survival (PFS) in VHL Disease-Associated RCC Tumors(Up to approximately 4 years)
- Time to Surgery (TTS) in VHL Disease-Associated RCC Tumors(Up to approximately 4 years)
- ORR in VHL Disease-Associated Non-RCC Tumors(Up to approximately 4 years)
- DOR in VHL Disease-Associated Non-RCC Tumors(Up to approximately 4 years)
- TTR in VHL Disease-Associated Non-RCC Tumors(Up to approximately 4 years)
- PFS in VHL Disease-Associated Non-RCC Tumors(Up to approximately 4 years)
- TTS in VHL Disease-Associated Non-RCC Tumors(Up to approximately 4 years)
- Number of Participants Experiencing an Adverse Event (AE)(Up to approximately 4 years)
- Number of Participants Experiencing a Serious Adverse Event (SAE)(Up to approximately 4 years)
- Belzutifan Plasma Concentration(Weeks 1 and 3: pre-dose, 2 and 5 hours post-dose, Week 5, 9, and 13: post-dose.)
- Belzutifan Metabolite Plasma Concentration(Weeks 1 and 3: pre-dose, 2 and 5 hours post-dose, Week 5, 9, and 13: post-dose.)