A Phase 2 Study of Bevacizumab for Chronic Bleeding and Iron Deficiency Anemia in Hereditary Hemorrhagic Telangiectasia (Trial Using Systemic Bevacizumab to Treat HHT, TrUST-HHT)
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 33
- 试验地点
- 2
- 主要终点
- Change in hematologic support score from pretreatment to maintenance
研究概览
简要总结
This research study is studying to see whether bevacizumab may treat chronic bleeding and iron deficiency anemia in Hereditary Hemorrhagic Telangiectasia (HHT).
Hereditary Hemorrhagic Telangiectasia (HHT) is a disorder that causes abnormal blood vessel formation. In HHT, there is a mutation in the TGF-β pathway, which results in an increase of vascular endothelial growth factor (VEGF) levels. An increase in VEGF levels can result in poorly formed blood vessels that have a higher rate of bleeding than normal blood vessels. Bevacizumab is designed to block VEGF activity. It is believed that targeting increased VEGF levels may be able to treat HHT.
This research study involves the following study drug:
- Bevacizumab
详细描述
In this research study, the investigators are studying the study drug, bevacizumab. Researchers would like to see whether bevacizumab effectively treats Hereditary Hemorrhagic Telangiectasia (HHT) and what side effects occur.
- The research study procedures include: screening for eligibility, pretreatment period, study treatment, end-of-study visit, and follow-up visit.
- Participants will be observed for 12 weeks and then receive study treatment for 24 weeks and will be followed for 30 days after ending study treatment.
- This research study involves the following study drug: Bevacizumab
- It is expected that about 33 people will take part in this research study.
This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational drug to learn whether the drug works in treating Hereditary Hemorrhagic Telangiectasia (HHT). "Investigational" means that the drug is being studied.
The U.S. Food and Drug Administration (FDA) has not approved bevacizumab for Hereditary Hemorrhagic Telangiectasia (HHT), but it has been approved for other uses.
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 —（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
Bevacizumab
-
The research study procedures include: screening for eligibility, pretreatment period, study treatment, end-of-study visit, and follow-up visit. Each period consists of 12 weeks, for a total of 36 weeks.
-
Pretreatment Period:Hematologic Support: iron transfusions and red cell transfusions as determined by study doctor.
-
Induction Period (first 3 months of bevacizumab treatment):
-
Hematologic Support: iron transfusions and red cell transfusions as determined by study doctor.
-
Bevacizumab: once every 2 weeks via intravenous infusion for up to 12 weeks.
-
Maintenance Period (second 3 months of bevacizumab treatment):
-
Hematologic Support: Iron transfusions and red cell transfusions as determined by study doctor.
-
Bevacizumab: once every 4 weeks via intravenous infusion for up to 12 weeks.
干预措施: Bevacizumab (Avastin®) (Drug)
结局指标
主要结局
Change in hematologic support score from pretreatment to maintenance
时间窗: 36 Weeks
The change in Hematologic Support Score (HSS) from pretreatment to maintenance will be evaluated with a paired t-test or a Wilcoxon signed-rank test, whichever is most appropriate for the distribution of the data. Presence of a statistically-significant difference (P\<0.05) will determine if the study achieves its primary outcome measure.
次要结局
- Average Maintenance Epistaxis Severity Score(36 Weeks)
- Difference in the individual patient mean hemoglobin(36 Weeks)
- Difference in the individual patient pRBC transfusion requirement(36 Weeks)
- Difference in the individual patient intravenous iron infusion requirement(36 Weeks)
- Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v5.0.(All patients will be evaluable for toxicity from the time of their first treatment with bevacizumab up to 36 weeks)
研究者
Hanny Al-Samkari, MD
Sponsor Investigator
Massachusetts General Hospital