AVAILABILITY OF GCRC RESOURCES FOR LONG TERM FOLLOW UP OF PARTICIPANTS IN GENE TRANSFER PROTOCOLS RELEASE DATE: January 28, 2004 NOTICE: NOT-RR-04-005 National Center for Research Resources (NCRR) (http://www.ncrr.nih.gov/) In 2002, three years after receiving retrovirally transduced stem cells, two subjects who were enrolled in a gene transfer research study developed T cell leukemia. These events highlighted the potential for serious adverse events to emerge long after the subject’s active involvement in the study . Gene transfer research investigators must confront these risks in order to maximize the protection of human subjects who participate in gene transfer research protocols and to help advance understanding of the underlying mechanisms involved. To address these concerns, the FDA now recommends that all subjects enrolled in gene transfer studies be followed for 15 years after administration of the study agent. FDA implements this recommendation by requiring gene transfer investigators to document their long-term follow-up plans in their IND applications. FDA specifically expects investigators to perform annual physical examinations of each subject for the first five years after administration of the study agent and, for the subsequent ten years, to ask subjects to complete a questionnaire on health status changes. Moreover, FDA expects investigators employing hematopoietic stem cells transduced by retroviral vectors to conduct certain laboratory studies semi-annually for five years and then annually for the following ten years to determine whether clonal cell populations have developed. Compliance with these recommendations is reasonably straightforward when the supporting grant is active. However, if the grant support ends, compliance becomes more challenging. Nonetheless, grantees and their institutions must make every effort to ensure that follow up studies of gene transfer study participants are carried out and the data submitted to the FDA. To facilitate NIH grantee and institutional compliance with the FDA requirements, the National Center for Research Resources (NCRR) will support the cost of subject visits at a General Clinical Research Center (GCRG) located either at the institution to which the award was made or elsewhere. The GCRC will provide clinical space, nursing, appointment scheduling, relevant routine laboratory tests, and phlebotomy. The grantee institution will be responsible for providing support for subjects to travel to the GCRC. The resources of the GCRCs will not be freely available to investigators of industry-sponsored studies. If GCRC resources are used in such instances, the institution will be responsible for obtaining reimbursement from the trial sponsor according to GCRC Guidelines http://www.ncrr.nih.gov/clinical/cr_gcrc.asp. If blood samples need to be collected and stored, they may be drawn by the GCRCs and stored at the National Gene Vector Laboratory (NGVL) repository at the Indiana University http://www.ngvl.org. Storage services will be provided at no cost to NIH funded clinical gene transfer protocols. Certain clonality tests may also be available at no cost to the investigators through the NGVL. The data gathered during the 15-year follow-up period is to be submitted to the FDA. There is no requirement for investigators to provide the data to the NIH funding institute unless the funding institute otherwise requires such data. Investigators must, however, continue to comply with reporting requirements outlined in the NIH Guidelines for Research Involving Recombinant DNA Molecules. Principal investigators who receive NIH awards for clinical gene transfer studies should ensure that during the consent process participants are fully informed about the FDA requirements and the processes that are involved in conducting the long-term follow up studies. INQUIRIES Direct your questions about NCRR GCRC issues to: Richard A. Knazek, M.D. Division of Clinical Research Resources National Center for Research Resources Democracy One, Room 910 6701 Democracy Blvd. Bethesda, Maryland 20892 Phone 301-435-0792 FAX 301-480-3661


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