Thymus Transplantation Safety-Efficacy
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|ClinicalTrials.gov Identifier: NCT01220531|
Recruitment Status : Available
First Posted : October 14, 2010
Last Update Posted : May 11, 2017
Complete DiGeorge anomaly (cDGA) is a disorder in which there is no thymus function. With no thymus function, bone marrow stem cells do not develop into T cells, which fight infection. Complete DiGeorge anomaly patients cannot fight infection and are immunodeficient. Without successful treatment, cDGA patients usually die by age 2 years.
Thymus transplantation with and without immunosuppression (drugs given before and after transplantation) has resulted in the development good T cell function in complete DiGeorge anomaly subjects.
This Phase I/II study continues thymus transplantation safety and efficacy research for the treatment of complete DiGeorge anomaly. Eligible participants undergo thymus transplantation and biopsy. Immune function testing is continued for one year post-transplantation.
|Condition or disease||Intervention/treatment|
|Complete DiGeorge Anomaly DiGeorge Syndrome DiGeorge Anomaly Complete DiGeorge Syndrome||Biological: Thymus Tissue for Transplantation Procedure: Blood Draw Drug: Rabbit anti-thymocyte globulin Drug: Cyclosporine Drug: Tacrolimus Drug: Methylprednisolone or Prednisolone Drug: Basiliximab Drug: Mycophenolate mofetil|
Complete DiGeorge anomaly (cDGA) is a congenital disorder characterized by athymia. Without successful treatment, children remain immunodeficient and usually die by age 2 years. In complete DiGeorge subjects, thymus transplantation with and without immunosuppression has resulted in diverse T cell development and good T cell function. The purpose of this Phase I/II study is to continue thymus transplantation safety and efficacy research for the treatment of complete DiGeorge anomaly. Until thymus transplantation is FDA approved as standard care for DiGeorge anomaly, research study participation is the only means by which a patient may have access to this potentially life-saving procedure.
This protocol includes 4 groups: one for subjects who do not require immunosuppression; and 3 immunosuppression groups for subjects with different T cell function levels to be suppressed adequately.
Eligible subjects undergo thymus transplantation and an allograft biopsy. Protocol specified studies continue until approximately one year post-transplantation.
Study participation lasts two years.
|Study Type :||Expanded Access|
|Official Title:||Safety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836|
|Study Start Date :||December 2010|
|Estimated Primary Completion Date :||December 2021|
|Estimated Study Completion Date :||December 2023|
Biological: Thymus Tissue for Transplantation
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01220531
|Contact: M. Louise Markert, M.D., Ph.Dfirstname.lastname@example.org|
|Contact: Stephanie Gupton, RN, CPNPemail@example.com|
|United States, North Carolina|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27701|
|Contact: M. Louise Markert, M.D., Ph.D 919-684-6263 firstname.lastname@example.org|
|Contact: Stephanie Gupton, RN, CPNP 919-684-4704 email@example.com|
|Principal Investigator: M. Louise Markert, M.D., Ph.D|
|Principal Investigator:||M. Louise Markert, M.D., Ph.D||Duke University Medical Center, Pediatrics, Allergy & Immunology|