A -- CLINICAL TRIALS AND CLINICAL MARKERS FOR IMMUNOLOGIC DISEASES Clinical Trials and Clinical Markers for Immunologic Diseases SOL RFP-NIH-NIAID-DAIT-BAA-99-12 POC Mr. Lawrence M. Butler, Contracting Officer, 301-496-0192. The National Institute of Allergy and Infectious Diseases intends to negotiate with Children's National Medical Center (CNMC), in order to test the hypothesis that treatment with an immunosuppressive preparative regimen utilizing fludarabine and ATG prior to stem cell transplantation can result in full or mixed donor cell chimerism and immune reconstitution in children with primary T-cell immune deficiencies who have traditionally had poor or variable engraftment when a preparative regiment has not been used. A second phase I/II trial in patients who have traditionally received fully myeloablative preparative regimens will utilize fludarabine and ATG with deescalating myelosuppressive doses of busulfan to determine the lowest myelosuppressive dose that results in engraftment. A retrospective study of patients with T-cell immune deficiencies who have already been transplanted will be performed to correlate preparative regimens with donor cell chimerism and immunologic function. Because of the rarity of and diversity of PID, a cooperative group format is mandatory for the study of any therapeutic intervention in these diseases. The proposed studies will be performed by CNMC under the auspices of the Pediatric Blood and Marrow Transplant Consortium, a consortium of over 65 transplant centers. This consortium has provided CNMC access to this unique population for the proposed studies to be conducted, therefore, CNMC is considered to be the only responsible source. Authority: 41 U.S.C. 253(c)(1), as set forth in FAR 6.302-1. See Numbered Note 22. Posted 05/12/00 (W-SN454440).

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