Hematopoietic stem cell transplantation (HCT) and cellular therapy offer curative therapy for a variety of malignant and non-malignant blood diseases. This is a rapidly evolving field with active preclinical and clinical development of new approaches to patient assessment, cell selection and manipulation, and pre- and post- treatment therapy. New diagnostic and therapeutic strategies require evaluation in definitive clinical trials as does the role of HCT versus other therapies. HCT and cellular therapy trials face unique challenges, including the relatively small number of procedures performed at any single center, the diverse indications for treatment requiring dissimilar approaches, the complex nature of the interventions, the risk of multiple complications in the immediate post-treatment period, and the risk of important, though infrequent, late effects. The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) was established in 2001 to develop and execute scientifically meritorious, prospective clinical trials to address these issues. The Network has launched 39 trials with the support of a Data and Coordinating Center (DCC) formed though a Consortium including the Center for International Blood and Marrow Transplant Research (CIBMTR) at the Medical College of Wisconsin, the National Marrow Donor Program, and The Emmes Corporation. This Consortium proposes to continue supporting the Network and managing the efficient development, implementation and completion of high- quality Phase I-III clinical trials, including concept evaluation and prioritization, protocol development with appropriate statistical designs, timely activation and accrual, monitoring for safety, compliance and data accuracy, and analyzing and disseminating results. The DCC Consortium will coordinate and support all BMT CTN activities maintaining a state-of-the-art database and systems for acquisition and storage of biospecimens; ensuring data quality, laboratory compliance and adherence to regulatory requirements; managing contractual arrangements and fiscal activities; monitoring and improving center and overall Network performance; and supporting all Network Committees and activities with meeting planning and other logistical support. The DCC will also continue to leverage Network resources and expertise through collaboration with other scientific bodies including the National Cancer Institute-funded National Clinical Trials Network Groups, NMDP's Be The Match BioTherapies, and CIBMTR's Working Committees and Stem Cell Therapeutic Outcomes Database, as well as industry partners. These collaborations are imperative to maximize completion of high quality and high priority HCT clinical trials, especially in the areas of HCT for non-malignant blood disorders and novel cell/gene therapies and which often must involve non-transplant investigators.
The proposed Data and Coordinating Center (DCC) will support all activities of the BMT CTN, using the extensive expertise of its investigators in clinical research, biostatistics and transplantation. The DCC will take advantage of collaborations with other organizations and initiatives to maximize efficiency and trial participation. The goal is to facilitate trials that provide definitive answers to questions and lead to improvements in the understanding and outcomes of hematopoietic cell transplantation and cellular therapies.
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