LIGHTBEAM-U01 Substudy 01E: A Phase 2 Substudy to Evaluate the Safety and Efficacy of Belzutifan in Participants With Solid Tumors
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 15
- 主要终点
- Number of Participants Who Experience One or More Adverse Events (AEs)
研究概览
简要总结
Researchers are looking for new ways to treat adolescents with locally advanced, unresectable, or metastatic solid tumors. Participants were enrolled into pheochromocytoma/paraganglioma (PPGL), wild type gastrointestinal stromal tumor (wtGIST), and Von Hippel-Lindau (VHL) disease-associated localized tumors cohorts:
- PPGL are rare cancers that start in cells that make hormones in the adrenal glands
- wtGIST is a less common type of cancer that starts in the digestive tract. Wild type means it does not have certain gene mutations (changes)
- VHL disease-associated localized tumors are rare tumors caused by a certain gene mutation that may be passed down from parents to children
- Locally advanced means the cancer has spread into nearby tissue
- Unresectable means the cancer cannot be removed by surgery
- Metastatic means the cancer has spread to other parts of the body
The goal of the study is to learn about the safety of belzutifan and if people tolerate it.
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
- 12 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The main inclusion criteria include but are not limited to the following:
- •Has a diagnosis of one of the following: locally advanced, unresectable, or metastatic pheochromocytoma/paraganglioma or wild-type gastrointestinal stromal tumors, or localized tumors associated with von Hippel-Lindau disease
- •Has measurable disease per RECIST 1.1
排除标准
- •The main exclusion criteria include but are not limited to the following:
- •Has a pulse oximeter reading <92% at rest, requires intermittent supplemental oxygen, or requires chronic supplemental oxygen
- •Has clinically significant cardiac disease or electrocardiogram indicating uncontrolled cardiac condition or has congenital long QT syndrome
- •Has a history of human immunodeficiency virus infection
- •Has received prior treatment with any hypoxia inducible factor-2α inhibitor, including belzutifan
- •Has had an allogenic tissue/solid organ transplant
- •Has a history of autologous stem cell transplant within 6 months of start of study intervention
- •Has known additional malignancy that is progressing or has required active treatment within the past 2 years
- •Has known active central nervous system metastases and/or carcinomatous meningitis
- •Has an active infection requiring systemic therapy
研究组 & 干预措施
Belzutifan
Participants will receive belzutifan 80 mg (body weight <40 kg) or 120 mg (body weight ≥40 kg) orally once daily for approximately 2 years.
干预措施: Belzutifan (Drug)
结局指标
主要结局
Number of Participants Who Experience One or More Adverse Events (AEs)
时间窗: Up to approximately 5 years
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The number of participants that experience AEs will be reported.
Number of Participants Who Discontinue Study Intervention Due to an AE
时间窗: Up to approximately 5 years
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a study intervention. The number of participants that discontinue study intervention due to an AE will be reported.
次要结局
- Area Under the Concentration-Time Curve From Time 0 to 24 hours of Belzutifan(At designated timepoints (up to 5 weeks))
- Minimum Plasma Concentration (Cmin) of Belzutifan(At designated timepoints (up to 5 weeks))
- Maximum Plasma Concentration (Cmax) of Belzutifan(At designated timepoints (up to 5 weeks))
- Objective Response Rate (ORR)(Up to approximately 5 years)
- Duration of Response (DOR)(Up to approximately 5 years)