A Phase I Study of Ipilimumab (Anti-CTLA-4) in Children, Adolescents, and Young Adults With Treatment Refractory Cancer
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 33
- 试验地点
- 3
- 主要终点
- To determine the tolerance and toxicity profile
研究概览
简要总结
This study will examine the safety and efficacy of ipilimumab-an experimental cancer treatment drug used to boost immune response-in children, adolescents, and young adults. Ipilimumab may allow immune cells to react to and destroy abnormal cells in the body, and has been tested in adults for a variety of cancers and has shown responses in some research studies. Because ipilimumab has not been tested in children, adolescents, or young adults, it is considered an experimental drug. The purposes of this research study are to determine the highest safe dose of ipilimumab for children, adolescents, and young adults with solid tumor cancers; examine its effectiveness and possible side effects; and better understand how the body and the immune system process it over time.
Candidates must be between 2 and 21 years of age and must have solid malignant tumors that have been resistant to standard therapy. Volunteers will be screened with a medical history, a clinical examination, and computerized scans such as magnetic resonance imaging (MRI). Participants must have completed their last dose of chemotherapy, radiation, chemotherapy, or antibody or investigational therapy at least four weeks prior to enrollment.
During the study, participants will receive an intravenous dose of ipilimumab once every three weeks. The infusion of ipilimumab will last 90 minutes, and the participant s vital signs will be monitored while the medicine is infusing and several times in the first 24 hours after the first dose (requiring a hospital stay during that time). If the participant is able to tolerate the first dose of ipilimumab, further doses (called cycles ) may be received on an outpatient basis. Blood and urine tests will be given on a regular basis during these cycles. After four cycles, participants whose tumors do not grow and who do not have unacceptable side effects will continue to receive ipilimumab every three months to maintain the current condition, until researchers conclude the study.
详细描述
Background:
Solid tumors represent approximately one fourth of cancer diagnoses in children. Despite intensive regimens, patients with metastatic or recurrent solid tumors have unsatisfactory survival rates. Therefore new therapies are needed to improve outcomes.
Accumulating preclinical and clinical evidence supports the use of biologic approaches to heighten antitumor immunity in order to improve the effectiveness of immune based therapy. Both directly activating immune based therapies such as cytokines and tumor vaccines as well as therapies which disrupt negative counterregulatory signals such as those mediated by CTLA-4:B7 may enhance existent antitumor immune responses.
Antibodies directed against CTLA-4 potently augment immune responses in animal models and anti-CTLA-4 antibodies have demonstrated antitumor effects in a variety of preclinical tumor models.
Phase I and phase II studies using ipilimumab have been performed in adults with a variety of tumor types. Clinical responses have been observed in renal cell carcinoma, melanoma, and prostate cancer. No trials have yet been performed to evaluate ipilimumab in children with malignancy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
1
Ipilumumab (DSE) given on day 1 of 21 day cycle for 4 cycles, from cycle 5+ ipilumumab will be given ~every 12wks
干预措施: Ipilimumab (Drug)
结局指标
主要结局
To determine the tolerance and toxicity profile
时间窗: 1 year
Pharmacokinetics
时间窗: 1 year
次要结局
- Quantify antitumor effects(2 years)
- Evaluate immunomodulatory activity.(2 years)