Phase I/II Trial of Thymus Transplantation With Immunosuppression, #950
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Survival at 1 Year Post-CTTI
研究概览
简要总结
The study purpose is to determine if cultured thymus tissue implantation (CTTI) (previously described as transplantation) with tailored immunosuppression based on the recipient's pre-implantation T cell population is a safe and effective treatment for complete DiGeorge anomaly. This study will also evaluate whether cultured thymus tissue implantation and parathyroid transplantation with immunosuppression is a safe and effective treatment for complete DiGeorge anomaly and hypoparathyroidism.
详细描述
Complete DiGeorge anomaly is a congenital disorder characterized by athymia. Without successful treatment, children remain immunodeficient and usually die by age 2 years. In infants with complete DiGeorge anomaly and no T cells, cultured thymus tissue implantation (CTTI) without immunosuppression resulted in diverse T cell development and good T cell function. Some infants with no thymus have some T cells that presumably developed extrathymically; these T cells can reject a thymus graft.
The purpose of this study is to tailor immunosuppression use for complete DiGeorge anomaly subjects who have some T cells and different T cell function levels. This protocol includes tailored immunosuppression regimens to allow subjects with different T cell function levels to be suppressed adequately.
Patients with complete DiGeorge often have hypoparathyroidism, a life threatening condition. Successful CTTI does not result in improvement of the hypoparathyroidism. The patients must go to the clinic for frequent calcium levels and to the hospital for calcium infusions. These infants are at risk for seizures from low calcium. This study had a parental parathyroid transplant arm for subjects with hypoparathyroidism who require calcium replacement.
Whether or not a subject was enrolled in the parathyroid arm, the immunosuppression regimen the subject received was dependent on the immune findings as stated in the clinical protocol.
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
- 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
Cultured Thymus Tissue Implantation (CTTI) w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergo cultured thymus tissue implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function.
干预措施: Cultured Thymus Tissue for Implantation (CTTI) (Biological)
Cultured Thymus Tissue Implantation (CTTI) w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergo cultured thymus tissue implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function.
干预措施: Blood Draw (Procedure)
Cultured Thymus Tissue Implantation (CTTI) w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergo cultured thymus tissue implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function.
干预措施: Rabbit anti-thymocyte globulin (Drug)
Cultured Thymus Tissue Implantation (CTTI) w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergo cultured thymus tissue implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function.
干预措施: Cyclosporine (Drug)
Cultured Thymus Tissue Implantation (CTTI) w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergo cultured thymus tissue implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function.
干预措施: Tacrolimus (Drug)
Cultured Thymus Tissue Implantation (CTTI) w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergo cultured thymus tissue implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function.
干预措施: Methylprednisolone or Prednisolone (Drug)
Cultured Thymus Tissue Implantation (CTTI) w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergo cultured thymus tissue implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function.
干预措施: Daclizumab (Drug)
Cultured Thymus Tissue Implantation (CTTI) w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergo cultured thymus tissue implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function.
干预措施: Mycophenolate mofetil (Drug)
CTTI with Parathyroid Transplantation w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergoes cultured thymus tissue thymus implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function. If the patient has hypoparathyroidism, and is eligible, the patient may also receive a parathyroid transplant.
干预措施: Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation (Other)
CTTI with Parathyroid Transplantation w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergoes cultured thymus tissue thymus implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function. If the patient has hypoparathyroidism, and is eligible, the patient may also receive a parathyroid transplant.
干预措施: Blood Draw (Procedure)
CTTI with Parathyroid Transplantation w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergoes cultured thymus tissue thymus implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function. If the patient has hypoparathyroidism, and is eligible, the patient may also receive a parathyroid transplant.
干预措施: Rabbit anti-thymocyte globulin (Drug)
CTTI with Parathyroid Transplantation w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergoes cultured thymus tissue thymus implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function. If the patient has hypoparathyroidism, and is eligible, the patient may also receive a parathyroid transplant.
干预措施: Cyclosporine (Drug)
CTTI with Parathyroid Transplantation w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergoes cultured thymus tissue thymus implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function. If the patient has hypoparathyroidism, and is eligible, the patient may also receive a parathyroid transplant.
干预措施: Tacrolimus (Drug)
CTTI with Parathyroid Transplantation w/immunosuppression
Patients with complete DiGeorge Anomaly (cDGA) undergoes cultured thymus tissue thymus implantation (previously described as transplantation) with tailored immunosuppression based on the subject's pre-implantation T cell numbers and function. If the patient has hypoparathyroidism, and is eligible, the patient may also receive a parathyroid transplant.
干预措施: Methylprednisolone or Prednisolone (Drug)
结局指标
主要结局
Survival at 1 Year Post-CTTI
时间窗: 1 year post-CTTI
Survival at 1 year post cultured thymus tissue implantation was assessed using the Kaplan Meier Estimated Survival. This mathematical function estimates the survival for a certain length of time.
次要结局
- Immune Reconstitution Efficacy - Naive CD8 T Cells(1 year post-CTTI)
- Immune Reconstitution Efficacy - Total CD3 T Cells(1 year post-CTTI)
- Immune Reconstitution Efficacy - Total CD8 T Cells(1 year post-CTTI)
- Immune Reconstitution Efficacy - Naive CD4 T Cells(1 year post-CTTI)
- Immune Reconstitution Efficacy - Response to Mitogens(1 year post-CTTI)
- Survival at 2 Years Post-CTTI(2 years post-CTTI)
- Immune Reconstitution Efficacy - Total CD4 T Cells(1 year post-CTTI)
- Thymus Allograft Biopsy(2 to 3 months post-CTTI)