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Thymus Transplantation Safety-Efficacy

Expanded access is currently available for this treatment.
Verified May 2017 by M. Louise Markert, Duke University Medical Center
Sponsor:
ClinicalTrials.gov Identifier:
NCT01220531
First Posted: October 14, 2010
Last Update Posted: May 11, 2017
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.
Collaborators:
National Institutes of Health (NIH)
National Institute of Allergy and Infectious Diseases (NIAID)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Enzyvant Therapeutics GmbH
Information provided by (Responsible Party):
M. Louise Markert, Duke University Medical Center
September 22, 2010
October 14, 2010
May 11, 2017
 
Thymus Transplantation Safety-Efficacy

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.

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.

Expanded Access
, Intermediate-size Population, Treatment IND/Protocol
  • 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 undergoes allograft biopsy. At the time of transplantation and biopsy, skin biopsy conducted. Subjects undergo laboratory testing for approximately one year post-transplantation. At year 2 post-transplantation, subjects are contacted for data collection.
    Other Name: Thymus Tissue Transplant
  • Procedure: Blood Draw
    Biological Mothers of Thymus Recipients are 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. 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. 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 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:
    • MMF
    • CellCept
 
Available
Contact: M. Louise Markert, M.D., Ph.D 919-684-6263 marke001@mc.duke.edu
Contact: Stephanie Gupton, RN, CPNP 919-684-4704 stephanie.gupton@dm.duke.edu
United States
 
 
NCT01220531
M. Louise Markert, Duke University Medical Center
M. Louise Markert
  • National Institutes of Health (NIH)
  • National Institute of Allergy and Infectious Diseases (NIAID)
  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  • Enzyvant Therapeutics GmbH
Principal Investigator: M. Louise Markert, M.D., Ph.D Duke University Medical Center, Pediatrics, Allergy & Immunology
Duke University
May 2017