Phase II Study of Danazol With Plasma Exchange and Steroids for the Treatment of Thrombotic Thrombocytopenic Purpura
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Number of Plasma Exchanges
研究概览
简要总结
The purpose of the study is to find out if administration of danazol with plasma exchange and corticosteroids will reduce the number of plasma exchanges required to control Thrombotic Thrombocytopenic Purpura (TTP).
详细描述
Danazol is a synthetic steroid hormone structurally resembling a group of natural hormones (androgens)found in the body. Danazol has immune modifying activity and is effective in treatment of blood disorders with low platelet counts such as idiopathic thrombocytopenic purpura (however FDA has not yet approved danazol for this disorder). A study of danazol in conjunction with plasma exchange for thrombotic thrombocytopenic purpura showed that danazol decreased the number of plasma exchanges required by approximately 80% and reduced the time needed to control the disease. It's not clear how danazol works in TTP. It is not approved by the FDA for the treatment of TTP.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 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
- •TTP with platelets less than 100,000 and microangiopathic hemolytic anemia
- •Age greater than 18 and less than 60
- •LDH > 2x upper limit of normal
- •PT and PTT normal
- •Patients must give signed informed consent
- •Pre-menopausal woman must have negative pregnancy test.
- •TTP not related to underlying cancer, treatment of cancer or transplantation.
- •TTP not associated with drugs.
排除标准
- •LFTs AST/ALT > 2x upper limit of normal
- •Hepatitis B and Hepatitis C infection.
- •HIV with active opportunistic infections
- •Acute or chronic Disseminated Intravascular Coagulation (DIC), defined as D-dimer 8ug/ml and fibrinogen<100 mg/dl
- •TTP related to drugs, malignancy and transplantation.
- •Pregnancy
- •Concurrent other investigational drug use during this study.
- •Porphyria.
研究组 & 干预措施
Danazol, Plex, Steroids
Everyone will receive Danazol with plasma exchange and corticosteroids
干预措施: Danazol (Drug)
结局指标
主要结局
Number of Plasma Exchanges
时间窗: up to 30 days
The total number of plasma exchanges performed after initiation of the first plasma exchange.
次要结局
- Number of Participants With Relapses(up to 12 years)
- Time to Remission(up to 30 days)
- Time to Relapse(up to 12 years)
- Number of Relapses(up to 12 years)
- Number of Participants With Complete and Continuous Response Rate(At 2 Years)
- Length of Stay(up to 30 days)
研究者
Ilan Shapira
Assistant Professor
Icahn School of Medicine at Mount Sinai