Parathyroid and Thymus Transplantation in DiGeorge #931

Summary

This study has three primary purposes: to assess parathyroid function after parathyroid transplantation in infants with Complete DiGeorge syndrome; to assess immune function development after transplantation; and, to assess safety and tolerability of the procedures. This is a Phase 1, single site, open, non-randomized clinical protocol. Enrollment is closed and study intervention is complete for all enrolled subjects; but subjects continue for observation and follow-up. Subjects under 2 years old with complete DiGeorge syndrome (atypical or typical) received thymus transplantation. Subjects received pre-transplant immune suppression with rabbit anti-human-thymocyte-globulin. Subjects with hypoparathyroidism and an eligible parental donor received thymus and parental parathyroid transplantation. A primary hypothesis: Thymus/Parathyroid transplant subjects will need less calcium and/or calcitriol supplementation at 1 year post-transplant as compared to historical controls.

Conditions

• DiGeorge Syndrome
• Hypoparathyroidism
• Complete DiGeorge Syndrome

Interventions

BIOLOGICAL

Thymus/Parathyroid Transplantation

Thymus tissue, thymus donor, mother of thymus donor, \& parental parathyroid donor screened for transplant safety. Depending on T cell phenotype \& function, subjects were given 1 of 2 immunosuppression regimes. All received rabbit anti thymocyte globulin pretransplantation. Others also received cyclosporine pre \& post-transplantation. The thymus dose was over 0.2 grams/kg recipient weight. Thymus transplant occurred in operating room; thymic slices were placed in quadriceps. Parathyroid harvest was done under general anesthesia. One parathyroid gland was minced and placed in quadriceps muscle. There was no dose in mg. An open biopsy of thymus allograft was done 2-3 months post-transplant. Biopsy tissue was examined by immunohistochemistry to evaluate for thymopoiesis \& graft rejection.

Sponsors & Collaborators

• Food and Drug Administration (FDA)

  collaborator FED

• National Institutes of Health (NIH)

  collaborator NIH

• National Institute of Allergy and Infectious Diseases (NIAID)

  collaborator NIH

• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

  collaborator NIH

• Sumitomo Pharma Switzerland GmbH

  lead INDUSTRY

Principal Investigators

• M. Louise Markert, MD, PhD · Duke University Medical Center, Pediatrics, Allergy & Immunology

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Max Age

24 Months

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2005-01-31

Primary Completion

2007-08-31

Completion

2019-12-31

FDA Drug

Yes

Countries

• United States

Study Locations