Reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan and anti-thymocyte globulin for chronic granulomatous disease: a multicenter phase II trial - CGD-RIST2

Sakaguchi Hirotoshi0 个研究点目标入组 22 人开始时间: 2019年3月20日最近更新: 2023年10月17日

适应症

试验速览

暂无简介。

•1) Diagnosed as chronic granulomatous disease
•2) Planned to have stem cell transplantation
•3) Donor for transplantation is one of the follows;
•a. HLA 5/6 (A, B, DR) or more matched related donor
•b. HLA 5/6 (A, B, DR) or more matched unrelated donor
•4) Age at transplantation <25 years
•5) ECOG Performance status: 0-2
•6) Sufficient organ function, as follows;
•a. T-Bil <= 1.5 mg/dl
•b. Serum creatinine <= 0.8 mg/dl (age <5y), <= 1.2 mg/dl (age 5-9y), <= 1.5 mg/dl (10y or older)
•c. Ejection fraction 45% or better, and QTfc <0.45 sec
•7) Obtained informed consent from a guardian of the patient

•1. Intracranial hemorrhage
•2. Severe psychological disorders
•3. Pregnant or suspected pregnancy
•4. Receiving interferon gamma therapy within 4 weeks of the start of conditioning therapy
•5. Not eligible for this study at the discretion of the investigator.

发起方

Sakaguchi Hirotoshi