Thymus Transplantation Safety-Efficacy
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|ClinicalTrials.gov Identifier: NCT01220531|
Expanded Access Status : Available
First Posted : October 14, 2010
Last Update Posted : July 19, 2019
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 expanded access study continues thymus transplantation safety and efficacy research for the treatment of complete DiGeorge anomaly. Eligible participants undergo thymus transplantation. 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 expanded access 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 complete 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 may undergo an allograft biopsy. Protocol specified studies continue until approximately one year post-transplantation.
Study participation lasts two years or until thymus recipients are asked to participate in a long-term follow-up study.
|Study Type :||Expanded Access|
|Expanded Access Type :||Intermediate-size Population, Treatment IND/Protocol|
|Official Title:||Safety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836|
- Biological: Thymus Tissue for Transplantation
Potential thymus recipient subjects are screened for eligibility. Thymus tissue (unrelated donor), donor, & donor's mother screened for safety. Thymus transplantation is done under general anesthesia in the operating room. Thymus tissue is transplanted into the subject's quadriceps. Two to three months post-transplantation, if medically stable, the subject may undergo allograft biopsy. At the time of transplantation and biopsy, skin biopsy may be conducted. Subjects undergo laboratory testing for approximately one-year post-transplantation. At approximately year 2 post-transplantation, subjects are contacted for data collection.Other Name: Thymus Tissue Transplant
- Procedure: Blood Draw
Biological Mothers of Thymus Recipients may be asked to participate in the study and undergo phlebotomy to allow for testing of T cell identity in the complete DiGeorge subjects. If blood is not obtainable, then a buccal swab may be done.Other Name: Venipuncture
- Drug: Rabbit anti-thymocyte globulin
Three doses of 2 mg/kg IV prior to thymus transplantation for immune suppression groups 2, 3, and 4. Each dose is given over 12 hours. RATGAM is usually given on days -5, -4, and -3 prior to thymus transplantation.Other Name: RATGAM
- Drug: Cyclosporine
Csa may be given every 8 to 12 hours orally or IV before and after thymus transplantation for immune suppression groups 3 and 4. The Csa dose is dependent on T cell numbers and the target CSA trough levels. Csa is weaned as per protocol.Other Name: Csa
- Drug: Tacrolimus
If unable to tolerate cyclosporine, then FK506 is given. FK506 may be given every 8 to 12 hours orally or IV before and after thymus transplantation. FK506 dose is dependent on T cell numbers and the target FK506 trough levels. FK506 is weaned as per protocol.Other Name: FK506
- Drug: Methylprednisolone or Prednisolone
Steroids IV or orally may be given before and/or after thymus transplantation. Administration and dosage depends on T cell numbers. Steroids are weaned as per protocol.Other Name: Steroids
- Drug: Basiliximab
A single dose of Basiliximab 5 mg/kg IV may be given. Administration of Basiliximab depends on T cell numbers and T cell activation. A single dose of Basiliximab may be given after the administration of rabbit anti-thymocyte globulin and before thymus transplantation. If Basiliximab is not given before thymus transplantation, and, depending on the T cell numbers and T cell activation, a single dose of Basiliximab may be given 3 to 5 days after thymus transplantation.Other Name: Simulect
- Drug: Mycophenolate mofetil
Mycophenolate mofetil (MMF) may be given if the T cell count remains elevated 5 days after thymus transplantation. If MMF is given, the dose is 15 mg/kg/dose every 8 hours IV or orally. MMF may be stopped at 35 days or continued for up to six months after thymus transplantation.Other Names:
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|