Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 7
- 试验地点
- 1
- 主要终点
- To Determine the Efficacy of This Allogeneic Transplant Approach in Reconstituting Normal Hematopoiesis and Reversing the Clinical Phenotype of CGD
研究概览
简要总结
Background:
- Chronic Granulomatous Disease (CGD) causes immune system problems. Treatment is usually a bone marrow transplant from a fully matched donor. Researchers want to try using partially matched donors for patients who do not have a fully matched donor available. The researchers will also use the drug cyclophosphamide to try to improve the outcomes when using a partially matched donor.
Objective:
- To learn the effectiveness of using cyclophosphamide with a transplant from a partially matched donor in treating CGD.
Eligibility:
- Recipients: age 2-65 with CGD with an ongoing infection that has not been cured by standard treatment and no fully matched donor available in an appropriate timeframe.
Design:
-
Recipients will:
-
be admitted to the hospital 2 weeks before transplant.
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be screened with blood and urine tests, breathing and heart health tests, X-rays, and/or magnetic resonance imaging. They may have a bone marrow aspiration and biopsy.
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meet with a social worker and dentist.
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get chemotherapy, radiation, and other medicines.
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get an intravenous (IV) catheter in their chest.
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have the transplant.
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get more medicines and standard supportive care.
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have blood drawn frequently.
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have to stay in the Washington, D.C. area for 3 months post-transplant.
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be followed closely for the first 6 months, and then less frequently for at least 5 years.
详细描述
Allogeneic transplant using HLA matched donors, both related and unrelated, has proven curative for patients with various immunodeficiencies, including those with ongoing infections. However donor availability remains a limiting factor in the application of this treatment modality. The use of haploidentical donors has in the past been fraught with a greater rate of complications related to both higher rates of GvHD and delayed immunorecovery. Newer transplant regimens appear to have diminished these risks and improved outcomes. We propose using a subablative conditioning regimen followed by post-transplant cyclophosphamide for patients with CGD who do not have an HLA matched donor but whose circumstances necessitate the use of a potentially curative, albeit high-risk treatment modality.
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
- 2 Years 至 65 Years（Child, 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
CGD Recipient
CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described
干预措施: Sirolimus (Drug)
CGD Recipient
CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described
干预措施: Donor peripheral blood stem cells. (Biological)
CGD Recipient
CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described
干预措施: Cyclophosphamide post transplant (Drug)
CGD Recipient
CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described
干预措施: Total body 200cGy (Radiation)
CGD Recipient
CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described
干预措施: Cyclophosphamide (Drug)
CGD Recipient
CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described
干预措施: Fludarabine (Drug)
CGD Recipient
CGD patients that will undergo haplo transplantation with post-transplant cyclophosphamide as described
干预措施: Busulfan (Drug)
结局指标
主要结局
To Determine the Efficacy of This Allogeneic Transplant Approach in Reconstituting Normal Hematopoiesis and Reversing the Clinical Phenotype of CGD
时间窗: 5 years
Patient will have donor chimerism of greater than 20% and resolution of infection or autoimmunity at end of follow up
次要结局
- To Determine the Safety of This Allogeneic HSCT Approach in Patients With CGD Including Transplant Related Toxicity, the Incidence of Acute and Chronic Graft-versus-host Disease, Immune Reconstitution, Overalland Disease-free Survival.(1 year post transplant)