BGJ398 for the Treatment of Tumor-Induced Osteomalacia
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Number of Participants With Complete Metabolic Remission After Stopping BGJ398
研究概览
简要总结
Background:
People with tumor-induced osteomalacia (TIO) have small tumors that may cause low blood phosphorus, weak muscles, bone pain, and broken bones. The tumors may be so small they are hard to find or impossible to remove. Researchers want to test a drug that may help treat TIO.
Objective:
To see how the drug BGJ398 affects people with tumor-induced osteomalacia.
Eligibility:
People ages 18-85 who are in NIH protocol 01-D-0184 and have TIO that cannot be found or easily removed
Design:
At every study visit, participants will have:
- Medical history
- Physical exam
- Blood and urine tests
- Questions about their health and fatigue
At the screening visit, participants will also have a heart and eye tests. They may have other tests to find their tumor.
The baseline visit will be a 1-week stay in the clinic. Participants will have the regular study tests, plus:
- Their first dose of the study drug capsules
- Blood and urine collected every 2-4 hours for 24 hours. A thin plastic tube will be inserted in a vein to collect blood.
- Heart and kidney ultrasounds
- Activities that test strength
- 6-minute walk test
Participants will take the study drug for six 1-month cycles. In each cycle, participants will:
- Take the study drug every day for 4 weeks.
- Have 1 visit. Participants will collect their urine for 24 hours and have their blood drawn. Participants will have the regular study tests and repeat some baseline tests.
- Have blood and urine tests at their local lab.
Participants will have 1 visit at the end of the last cycle and another 3 months later....
详细描述
Background:
- Tumor-induced osteomalacia (TIO) is a rare disorder in which fibroblast growth factor (FGF23)-producing neoplasms cause renal phosphate wasting and skeletal disease.
- Recent studies have shown that chromosomal translocations causing a fibronectin-FGFR1 (FN1/FGFR1) fusion gene have been identified in 40-60% of these tumors.
- BGJ398 is an orally bio-available, selective and ATP competitive pan-fibroblast growth factor receptor (FGFR) kinase inhibitor which has demonstrated anti-tumor activity in preclinical, in vitro and in-vivo tumor models harboring FGFR genetic alterations.
Objectives:
To induce complete metabolic response in subjects with tumor-induced osteomalacia (TIO) with BGJ-398 as demonstrated by normalization of FGF23 and phosphate homeostasis.
Eligibility:
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
- 18 Years 至 85 Years（Adult, Older 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
Single Arm (TIO Subjects)
Phase 2, open-label, non-randomized, single-arm, drug treatment trial. 10 subjects will be studied. Treatment duration of 6 months with 3 months off drug follow-up and optional extension phase.
干预措施: BGJ398 (Drug)
结局指标
主要结局
Number of Participants With Complete Metabolic Remission After Stopping BGJ398
时间窗: Up to 12 weeks after stopping BGJ398
Participants were monitored for up to 12 weeks after stopping BGJ398. A participant was considered to have a complete metabolic remission by achieving both normal blood FGF23 and phosphorus levels in the blood for 12 weeks after stopping BGJ398.
次要结局
- Number of Participants With Grade 3 or 4 Adverse Events or Serious Adverse Events(24 week treatment phase followed by 3 month follow up or extension phase)
- Hand Grip Strength Test(Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)
- Number of Participants With Complete and Partial Metabolic Response Rate(Every 2 weeks up to 24 weeks)
- Pinch Test(Assessed at baseline and every 4 weeks for the 24 treatment period, and every 4 weeks in the follow up phase, up to 37 weeks)
- PROMIS Mobility Score(Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)
- Blood C-terminal FGF23 Level(Assessed at baseline and every 2 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)
- Blood 1,25-(OH)2-Vitamin D Level(Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)
- Six-Minute Walk Test(Assessed at baseline and 24 weeks after starting BGJ398)
- Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)(Assessed at baseline and every 2 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)
- Five Times Sit-to-Stand Test(Assessed at baseline and every 4 weeks during the 24 week treatment period)
- PROMIS Fatigue(Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)
- PROMIS Pain Interference Score(Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)
- Blood Intact FGF23 Levels(Assessed at baseline and every 2 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)
- Radiographic Evidence of Tumor-Induced Osteomalacia(Baseline and at 24 weeks)
- The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH)(Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)
- Blood Alkaline Phosphatase(Assessed at baseline and every 2 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)
- Tubular Reabsorption of Phosphate(Assessed at baseline and every 2 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)
- RAND SF-36 Survey Results(Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)
- Blood Phosphate Levels(Assessed at baseline and every 2 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks)