Human Clinical Core (CINRG)

The Human Clinical Core is a proposed "Scientific Research Resource Core". The overall theme of the Center application is to both identify and target specific pathways involved in the presentation, progression, and response to steroids of Duchenne muscular dystrophy, using both human patients with defined diagnoses, and corresponding murine models. The primary goal of the Human Clinical Core is to provide an infrastructure capable of supporting a continuum of well-designed clinical trials in a geographically dispersed network of clinics so that they are widely available to children with Duchenne muscular dystrophy (DMD) and other neuromuscular diseases. In addition, the Core will ensure appropriate IRB approvals, consent forms, research genetic counseling services, and interactions with the CNMC Clinical Research Center (CRC). We have made substantial strides in human muscular dystrophy clinical trials, through our Cooperative International Neuromuscular Research Group (CINRG) network. As a potential Scientific Research Resource Core, the long-term goal of this Core and our existing CINRG network is to enable a large number of newly diagnosed DMD children to be entered into one of a number of parallel clinical trials; currently 5 drug trials are underway, each supported by the proposed Core. Three of these pre-existing clinical trials form the basis for patient recruitment for the SNP association studies in Project 1. In developing our clinical trial organization, we have endeavored to promote a spirit of cross-disciplinary and cross-institutional collaboration in the field of pediatric neuromuscular disease. Over the last 3 years, we have achieved this by coordinating the efforts of basic, translational, and clinical researchers in DMD, by centralizing many of the key administrative and regulatory tasks within our core, and by taking advantage of both emerging and mature internet-based clinical trial technologies. The specific aims of this Core are largely focused on the CINRG clinical trial network infrastructure, and support of Project 1 (Dr. Hoffman). However, in the increasingly complex issues facing human clinical research, particularly involving children, this Core will also coordinate and centralize clinical research involving human subjects for Projects 2 and 3, ensuring that all existing and emerging regulations are diligently and appropriately adhered to (e.g. newly released HIPPA guidelines concerning confidentiality and sample storage)