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Pilot Study of Allogeneic Bone Marrow Transplantation Plus Cyclosporine and Mycophenolate Mofetil to Induce Mixed Hematopoietic Chimerism in Patients With Primary T-Cell Immunodeficiency Disorders

2014-07-23 21:56:13 | BioPortfolio

Summary

OBJECTIVES: I. Determine the safety of cyclosporine and mycophenolate mofetil as a non-ablative conditioning and post-transplantation immunosuppression regimen in patients with primary T-cell immunodeficiency disorders who undergo HLA-matched related or unrelated bone marrow transplantation to induce mixed hematopoietic chimerism (establishment of 1-95% donor CD3+ cells).

II. Determine the kinetics of immune reconstitution of lymphoid cell subsets, T-cell function, and B-cell function after allogeneic bone marrow transplantation in this patient population.

Description

PROTOCOL OUTLINE: Patients are stratified according to type of primary T-cell immunodeficiency disorder (severe combined immunodeficiency syndrome (SCID) vs non-SCID) and donor status (related vs unrelated).

All patients receive cyclosporine orally or IV on days -1 through 50 and oral mycophenolate mofetil on days 0 through 27 in the absence of unacceptable toxicity. Unrelated donor recipients and non-SCID patients also undergo total body irradiation on day 0. All patients then undergo allogeneic bone marrow transplantation on day 0. Cyclosporine taper regimen begins on day 50 and continues through day 180 unless evidence of graft-versus-host disease.

Study Design

Allocation: Non-Randomized, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Purine-Pyrimidine Metabolism, Inborn Errors

Intervention

Cyclosporine, Mycophenolate mofetil

Location

Fred Hutchinson Cancer Research Center
Seattle
Washington
United States
98109

Status

Recruiting

Source

Office of Rare Diseases (ORD)

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-07-23T21:56:13-0400

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