Thymus Transplantation Safety-Efficacy
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01220531|
Expanded Access Status : Available
First Posted : October 14, 2010
Last Update Posted : August 25, 2021
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 educated T cells, which fight infection. Without successful treatment, patients with cDGA must remain in reverse isolation to prevent infection and subsequent death.
Cultured thymus tissue with and without immunosuppression (drugs given before and after implantation) has resulted in the development of good T cell function in subjects with complete DiGeorge anomaly.
This expanded access study continues cultured thymus tissue safety and efficacy research for the treatment of complete DiGeorge anomaly. Eligible participants receive cultured thymus tissue. Immune function testing is continued for one year post-implantation.
|Condition or disease||Intervention/treatment|
|Complete DiGeorge Anomaly DiGeorge Syndrome DiGeorge Anomaly Complete DiGeorge Syndrome||Biological: Cultured Thymus Tissue Procedure: Blood Draw Drug: Rabbit anti-thymocyte globulin Drug: Cyclosporine Drug: Tacrolimus Drug: Methylprednisolone or Prednisolone Drug: Mycophenolate mofetil|
Complete DiGeorge anomaly (cDGA) is a congenital disorder characterized by athymia. Without successful treatment, patients with cDGA must remain in reverse isolation to prevent infection and subsequent death. In patients with cDGA, implantation of cultured thymus tissue 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 cultured thymus tissue safety and efficacy research for the treatment of athymia in patients with cDGA. Until administration of cultured thymus tissue is FDA approved as standard care for cDGA, 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 receive cultured thymus tissue and may undergo an allograft biopsy.
Protocol specified studies continue until approximately one year post-implantation. Study participation lasts two years.
|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: Cultured Thymus Tissue
Potential recipients of cultured thymus tissue are screened for eligibility. The thymus tissue (from an unrelated donor), the donor, and the donor's mother are screened for safety. Cultured thymus tissue is implanted under general anesthesia in the operating room. Cultured thymus tissue is placed into the subject's quadriceps. Two to three months post-implantation, if medically stable, subjects may undergo an allograft biopsy. Subjects undergo laboratory testing for approximately one-year post-implantation. At approximately year 2 post-implantation, subjects are contacted for data collection.Other Name: Thymus Tissue Transplant
- Procedure: Blood Draw
Other Name: Venipuncture
- Drug: Rabbit anti-thymocyte globulin
Three IV doses of 2 mg/kg RATGAM are given prior to implantation of cultured thymus tissue 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 implantation of cultured thymus tissue.Other Name: RATGAM
- Drug: Cyclosporine
Csa may be given every 8 or every 12 hours orally or IV before and after implantation of cultured thymus tissue 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 or every 12 hours orally or IV before and after implantation of cultured thymus tissue. 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 implantation of cultured thymus tissue. Steroid administration and dosage depends on T cell numbers. Steroids are weaned as per protocol.Other Name: Steroids
- Drug: Mycophenolate mofetil
Mycophenolate mofetil (MMF) may be given if the T cell count remains elevated 5 days after implantation of cultured thymus tissue. 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 implantation of cultured thymus tissue.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.Demail@example.com|
|Contact: Stephanie Gupton, RN, CPNPfirstname.lastname@example.org|
|United States, North Carolina|
|Duke University Medical Center||Available|
|Durham, North Carolina, United States, 27710|
|Contact: M. Louise Markert, M.D., Ph.D 919-475-6011 email@example.com|
|Contact: Stephanie Gupton, RN, CPNP 919-684-4704 firstname.lastname@example.org|
|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|