A Phase II Study of Pegylated Interferon Alfa-2b (Peg-Intron (TM)) in Children With Diffuse Pontine Gliomas
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 32
- 试验地点
- 2
- 主要终点
- Two Year Survival of Pediatric Patients With Diffuse Pontine Gliomas
研究概览
简要总结
Diffuse pontine gliomas are tumors on the pons portion of the brainstem. Their peak incidence is in children between 5 and 10 years old. Their location makes surgical resection impossible. Most patients are treated with radiation, which typically delays progression of the tumor for a median time of only about 6 months; median survival time is less than 1 year. The addition of chemotherapy has not improved the outcome.
Alpha, beta, and gamma interferons have been used to treat malignant brain tumors, with mixed results. Different doses and different methods of administration have been studied. Alpha interferon is usually given in high doses 2 or 3 times a week, but it has serious side effects at these doses. Recent studies have shown that administering chemotherapy more frequently at smaller doses (metronomic) may have a better effect against the tumor.
PEG-Intron(Trademark) is a form of interferon alpha combined with monomethoxy polyethylene glycol (PEG). It has a longer half-life than interferon alone, is administered once a week, and the long half-life reduces the peaks and troughs in blood levels.
This study will enroll 32 patients under age 21. The primary goals of the study are to determine if there is a difference in the 2-year survival rate of patients treated with radiation alone and those treated with radiation followed by PEG-Intron(Trademark) and to define the toxicities of PEG-Intron(Trademark) in the study doses. Secondary goals are to evaluate various magnetic resonance imaging (MRI) techniques for noninvasive monitoring of changes in the brainstem and to evaluate neuropsychological function.
In this study, PEG-Intron(Trademark) will be administered subcutaneously once a week at low doses (0.3 microgram per kilogram of body weight) for a 4-week cycle. The cycles will be repeated indefinitely until progression of disease or serious side effects develop. For less severe effects, the dose will be lowered and the patient may remain in the study. For more severe effects, the dose will be discontinued. Patients in the study may receive supportive medication but may not receive other forms of chemotherapy.
Patients or their caregivers will be instructed in how to inject the drug. Patients and/or caregivers will be asked to maintain a diary documenting the dose, site of administration, and any side effects. The diary will be reviewed at each National Cancer Institute (NCI) visit. Patients will return to NCI before cycles 2 and 3. If there are no significant side effects, patients may then return to NCI before every other cycle, indefinitely (i.e., before cycles 5, 7, 9, etc.).
Patients will undergo the following tests and procedures:
- Physical examination, including neurologic exam, monthly
- Complete blood count, differential, and platelet count weekly during cycle 1 and every 2 weeks thereafter if no severe side effects occur
- Blood chemistries weekly during cycle 1 and every 2 weeks thereafter if no severe side effects occur
- Endocrine function tests before each cycle
- Urinalysis before each cycle
- MRI of the brain before cycles 1, 2, 3, 5, 7, and every other month; patients may also have proton magnetic spectroscopic imaging performed at the time of the MRI
详细描述
Background:
Children with diffuse pontine gliomas have a dismal prognosis. Because surgery in this area is difficult, radiation therapy has been the mainstay of treatment. Although some children may improve clinically after radiation therapy, the effect is short-lived and almost all progress within several months. Chemotherapy has not had a significant impact on survival. Interferon-alpha is a cytokine that has been studied in patients with gliomas and has demonstrated some activity in prior clinical trials.
Objectives:
- To compare the 2-year survival of pediatric patients with diffuse pontine gliomas receiving weekly subcutaneous low-dose pegylated interferon alfa-2b (PEG-Intron[TM]) injections after standard radiation therapy versus historical controls who have received radiation therapy alone.
- To define the toxicities of weekly low-dose pegylated interferon alfa-2b (PEG-Intron(Trademark)) in pediatric patients.
Eligibility:
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
- — 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
结局指标
主要结局
Two Year Survival of Pediatric Patients With Diffuse Pontine Gliomas
时间窗: 8 yrs 6 mo 0 days
Survival is measured from the date the patient is registered onto the protocol until the day of death and the date of diagnosis to the date of patient death.
次要结局
- Median Time to Progression(8 yrs 11 mo 22 days)
- Number of Participants With Adverse Events(8 yrs 11 mo 22 days)
- Mean Quality of Life (QOL) Score at Baseline and Follow-Up(once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.)
- Number of Participants With a Metabolic and Biological Change in the Brainstem Through Magnetic Resonance Imaging (MRI) Techniques(once a week for 4 weeks beginning 2-10 weeks after completion of radiation therapy and continued until disease progression or one of the other off study criteria.)