PLA General Hospital
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- FVIII/FIX Activity
研究概览
简要总结
The goal of this clinical trial is to determine the clinical efficacy and toxic effects of sodium valproate, sirolimus and calcitriol in the treatment of severe haemophilia in participants with severe haemophilia . The main questions it aims to answer are the possibility of adding a combination regimen to primary treatment for severe haemophilia . Patients will receive oral sodium valproate extended-release tablets 0.5g/day, sirolimus tablets 1mg/day and osteopontin capsules 0.25μg/day.
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
- 14 Years 至 85 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with clinically confirmed severe haemophilia;
- •Expected survival of ≥ 24 weeks with an ECOG score of 0-2;
- •Not having participated in another clinical trial within four weeks;
- •Informed consent signed by the patient or an immediate family member.
排除标准
- •Those with other types of blood disorders diagnosed at the morphological or molecular level of the bone marrow;
- •Significantly abnormal cardiopulmonary function;
- •Hepatic or renal insufficiency;
- •Pregnancy or lactation, or inability to use contraception during the trial and for three months before the test and one year after administration
- •Persons who are allergic to the drugs likely to be used or where there is a contraindication to their use;
- •Those with severe uncontrollable infectious diseases or uncontrolled hypertension, malignancy, etc.;
- •Inability to cooperate with a regular follow-up due to psychological, social, family and other geographical circumstances;
- •Any other condition that, in the investigator's opinion, makes participation in this trial inappropriate.
研究组 & 干预措施
Patients with severe haemophilia
confirmed as hemophilia and FVIII/FIX activity <1%
干预措施: Sodium valproate extended-release tablets (Drug)
结局指标
主要结局
FVIII/FIX Activity
时间窗: through study completion, an average of 1 month
FVIII/FIX activity in peripheral blood
FVIII/ FIX inhibitor concentration
时间窗: through study completion, an average of 1 month
FVIII/ FIX inhibitor concentration in peripheral blood
次要结局
- Activated Partial Thromboplastin Time(through study completion, an average of 1 month)
- frequency of joint bleeding(through study completion, an average of 1 month)
研究者
Xue-chun Lu
Director of Hematology Department of the Second Medical Center of PLA General Hospital
Chinese PLA General Hospital