Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 62
- 试验地点
- 1
- 主要终点
- Incidence of Graft versus Host Disease (GvHD)
研究概览
简要总结
A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- — 至 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Sickle Cell Disease (SCD)
- •SCD Patients with a fully matched sibling donor (MSD) irrespective of the frequency or severity of symptoms MSD transplant can be considered. Parents/patient must be counseled as to the risks and benefits and provide their voluntary informed consent
- •Transfusion Dependent Alpha- or Beta- Thalassemia
- •Diamond Blackfan Anemia
- •Other Non-Malignant Hematologic Disorders
- •Karnofsky ≥ 60%, Lansky play score ≥
- •Patients with lower performance score can be considered based on study team's evaluation.
- •Sexually active persons of childbearing potential or persons with partners of childbearing potential must agree to use a highly effective form of contraception during study treatment and for at least 4 months after the transplant.
排除标准
- •Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 30 days of the start of treatment
- •HIV infection with a detectable viral load. All HIV+ patients must be evaluated by infectious disease (ID) and an HIV management plan established prior to transplantation.
- •Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted
- •Known allergy to any of the study components
- •Psychiatric illness/social situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements
- •Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study
研究组 & 干预措施
Arm B
Arm B Matched sib regimen - 0-55 (per physician preference for patients over 6) ATG/Flu/Bu
干预措施: Cell Infusion (Biological)
Arm C
Arm C Fully Matched unrelated donor (MUD)- - 0-55 years; ATG/Flu/Bu
干预措施: Cell Infusion (Biological)
Arm B
Arm B Matched sib regimen - 0-55 (per physician preference for patients over 6) ATG/Flu/Bu
干预措施: Tacrolimus (Drug)
Arm D
Arm D: Haploindentical or mismatched unrelated donors (MMUD) - 0-55 years; ATG/Thiotepa/Cyclophosphamide/MESNA/Flu/TBI
干预措施: Sirolimus (Drug)
Arm B
Arm B Matched sib regimen - 0-55 (per physician preference for patients over 6) ATG/Flu/Bu
干预措施: Thymoglobulin (Drug)
Arm B
Arm B Matched sib regimen - 0-55 (per physician preference for patients over 6) ATG/Flu/Bu
干预措施: Fludarabine (Drug)
Arm B
Arm B Matched sib regimen - 0-55 (per physician preference for patients over 6) ATG/Flu/Bu
干预措施: Busulfan (Drug)
Arm B
Arm B Matched sib regimen - 0-55 (per physician preference for patients over 6) ATG/Flu/Bu
干预措施: Mycophenolate Mofetil (Drug)
Arm C
Arm C Fully Matched unrelated donor (MUD)- - 0-55 years; ATG/Flu/Bu
干预措施: Thymoglobulin (Drug)
Arm C
Arm C Fully Matched unrelated donor (MUD)- - 0-55 years; ATG/Flu/Bu
干预措施: Fludarabine (Drug)
Arm C
Arm C Fully Matched unrelated donor (MUD)- - 0-55 years; ATG/Flu/Bu
干预措施: Busulfan (Drug)
Arm C
Arm C Fully Matched unrelated donor (MUD)- - 0-55 years; ATG/Flu/Bu
干预措施: Tacrolimus (Drug)
Arm C
Arm C Fully Matched unrelated donor (MUD)- - 0-55 years; ATG/Flu/Bu
干预措施: Mycophenolate Mofetil (Drug)
结局指标
主要结局
Incidence of Graft versus Host Disease (GvHD)
时间窗: 1 year
次要结局
- Overall Survival(1 and 2 years)
- Grade 3-4 Acute GvHD(2 years)
- Chronic Graft versus Host Disease (GvHD) Free(2 years)
- Failure Free Survival(2 years)