A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Vestibular schwannoma volume
研究概览
简要总结
The purpose of the study is to determine if RAD001 treatment will shrink or slow the growth of the vestibular schwannoma(s) in Neurofibromatosis 2 (NF2) patients. Secondary objectives include determining if RAD001 treatment will improve hearing ability in NF2 patients.
RAD001 is an oral drug that is approved by Food and Drug Administration (FDA) for other types of tumors, it is not approved by the FDA for treatment of NF2 related tumors.
详细描述
This protocol is a Phase II, open-label, efficacy and safety study of single-agent RAD001 in patients with NF2. During the study, subjects will receive continuous daily oral treatment with RAD001 for up to 1 year or until tumor progression.
Primary Objective: To determine whether RAD001 has an effect on the VS growth in patients with NF2 at a rate sufficient to submit the drug for further testing.
Secondary Objectives: To determine whether RAD001 has an effect on the volume of other intracranial tumors, and to assess the effect of RAD001 on hearing function in patients with NF2 (when applicable).
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
- 16 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of NF2 by National Institutes of Health (NIH) criteria
- •Age ≥ 16 years
- •Progressive VS growth during the previous 12 months.
- •WHO performance status > or = 2
- •Adequate bone marrow, liver and renal function.
- •For women of childbearing potential, no pregnancy or breast-feeding
- •Willingness and ability to comply with scheduled visits, drug administration plan, laboratory tests, other study procedures, and study restrictions.
- •Willingness to provide informed consent
排除标准
- •Inability to tolerate periodic MRI scans or gadolinium contrast.
- •Inability to tolerate periodic audiologic testing or to understand a language with established scoring for word recognition testing.
- •Inability to adequately perform volumetric measurement of at least 1 target lesionNote: Patients with cochlear or auditory brainstem implants may participate if a target lesion can be accurately assessed.
- •Radiation therapy for the target lesion in the 60 months preceding inclusion in the study.
- •Patients currently receiving anticancer therapies or who have received anticancer therapies within 4 weeks of the start of study drug.
- •Immunization with attenuated live vaccines within one week of study entry or during study period.
- •Presence of a fungal infection requiring systemic antifungal treatment at enrollment
- •Other malignancies within the past 3 years except for adequately treated carcinoma of the cervix or basal or squamous cell carcinomas of the skin.
- •Patients who have any severe and/or uncontrolled medical conditions.
- •Patients with a known hypersensitivity to everolimus or other types of rapamycin or to its excipients.
- •Patients unwilling to or unable to comply with the protocol
研究组 & 干预措施
RAD001 Treatment
All subjects will be given RAD001 for 1 year (12 months).
干预措施: RAD001, everolimus (Drug)
结局指标
主要结局
Vestibular schwannoma volume
时间窗: 1 year (12 months)
Determine the effect of RAD001 on change in vestibular schwannoma volume (mm3) by MRI from baseline to 1 year.
次要结局
- Hearing(1 year (12 months))
- Number of adverse events(1 year, 1 month (13 months))