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Serum-Free Thymus Transplantation in DiGeorge Anomaly

NCT00849888 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2022-04-01

No results posted yet for this study

Summary

The study purpose is to determine if thymus tissue cultured in a serum-free (SF) solution is a safe and effective treatment for atypical and typical complete DiGeorge anomaly. \[Funding Source - FDA OOPD\]

Conditions

  • DiGeorge Anomaly

Interventions

BIOLOGICAL

Serum Free Thymus Transplantation with Immunosuppression

Cyclosporine pre-transplant (trough 180-220ng/ml) until naive T cells develop. Subjects \>4,000/cumm T cells, pre-transplant methylprednisolone or prednisolone 1-2mg/kg/day. All subjects pre-transplant days -5,-4,-3: 3 doses 2mg/kg rabbit anti-thymocyte globulin. Thymus tissue (unrelated donor), donor, \& donor's mother screened for safety. Transplant under general anesthesia into quadriceps. First 2 subjects, FBS cultured thymus is transplanted in 1 leg \& serum free (SF) in other. After first 2 subjects \>10% naïve T cells, 3rd receives only SF thymus. After 3rd subject \>10%naive T cells, 4th subject transplanted. Thymus dose 4-18 grams/m2 body surface area. Thymus biopsy 8-12 weeks post-transplant. Skin biopsy at time of transplant \& thymus biopsy. Followed by immune evaluations.

OTHER

Serum Free Thymus Transplantation without immunosuppression

Thymus tissue (unrelated donor), donor, \& donor's mother screened for safety. Transplant under general anesthesia into quadriceps. First 2 subjects: FBS cultured thymus transplanted in 1 leg \& serum free cultured thymus in other leg. After first 2 subjects have thymopoiesis in serum-free biopsy, \>10% naïve T cells, 3rd subject receives only serum free cultured thymus. After 3rd subject \>10% naive T cells, 4th subject receives transplant of only serum free cultured thymus. Dose 4-18grams/m2 body surface area. At time of transplant, skin biopsy. Allograft biopsy \& skin biopsy done 8 to 12 weeks post-transplant. (Graft biopsy not done if subject medically unstable.) Post-transplant, subjects followed by immune evaluations, using blood samples, for two years.

Sponsors & Collaborators

  • 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, M.D., Ph.D · Duke University Medical Center, Pediatrics, Allergy & Immunology

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Max Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-04-30
Primary Completion
2011-02-28
Completion
2011-02-28

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00849888 on ClinicalTrials.gov