A Phase I/II Open Label Individual Dose Titration Trial of the Human Corticotropin- Releasing Factor (hCRF), Corticorelin Acetate Injection (Xerecept®), to Determine the Tolerability of Xerecept® in a Pediatric Population
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- 入组人数
- 15
- 试验地点
- 2
- 主要终点
- Maximum Tolerated Dose
研究概览
简要总结
This study is being conducted to evaluate the safety and tolerability of Xerecept® in children with central nervous system tumors and to identify appropriate doses of Xerecept® to be used in subsequent pediatric clinical trials.
详细描述
Steroid-related side effects are much more severe in a pediatric population than they are in adults. The side effects appear more quickly and are often apparent in as few as 14 days. Dexamethasone side effects in children include rounding of the face, acne, increased body hair, muscle weakness, osteoporosis, mood changes, weight gain, fluid retention, glucose instability, high blood pressure, increased susceptibility to infection, stunted growth and aseptic necrosis of the hip joints14-16.
Although extensive studies have been performed with Xerecept® in adults, no studies have been performed with Xerecept® in pediatric subjects. Therefore, this study is being conducted to evaluate the safety and tolerability of Xerecept® in children with central nervous system tumors and to identify appropriate doses of Xerecept® to be used in subsequent pediatric clinical trials. The study will also explore if Xerecept®, when administered to pediatric patients, is effective in allowing decreases in dexamethasone dosing
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
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have histologically confirmed primary, recurrent or metastatic cerebral tumors and require chronic steroid dosing to manage symptoms of peritumoral brain edema (No histological confirmation is required of optic pathway tumors or brain stem gliomas)
- •Have been taking dexamethasone (or equivalent) at a dose of ≥1 mg/day for a minimum of 14 days and have had a stable dose for at least 7 days.
- •Have one or more steroid-associated side effects at Screening (See Appendix A1 for a list of qualifying events)
- •Capable of self-administration of subcutaneous injections twice daily or availability of assistance from caregiver.
- •Karnofsky/Lansky performance status ≥ 40
- •Life expectancy of at least 6 months
- •Female subjects must not be pregnant or breast-feeding and, if of childbearing potential, must use adequate methods of contraception during the study
- •Ability to provide written informed consent or, if unable to provide, have a legal guardian or representative provide written informed
排除标准
- •Evidence of previous or current overt renal, hepatic or pulmonary diseases or active uncontrolled infection
- •Subject and/or parent/guardian is unwilling or unable to comply with this protocol
- •Subject has a known history of allergy or intolerance to hCRF or other ingredients in the Xerecept® solution (e.g., methionine, mannitol)
- •Subject has a known history of hepatitis C virus, hepatitis B surface, or human immunodeficiency virus (HIV).
研究组 & 干预措施
Xerecept
All patients will receive hCRF (XERECEPT)
干预措施: XERECEPT (Drug)
结局指标
主要结局
Maximum Tolerated Dose
时间窗: 2 months
To define the maximum tolerated dose (MTD) of Xerecept® in pediatric patients; the MTD will be defined as the dose at which 1 or fewer of six patients experiences a dose limiting adverse event of any kind.
次要结局
- Dexamethasone Dosing(1 Year)
- Incidence and severity of specified Steroid-Related Side Effects(1 year)
- Number of patients with adverse events(1 Year)
- Change from baseline in clinical chemistry, hematology and urinalysis measures(1 Year)
- PedsQL™ Quality of Life Inventory Scores(1 Year)