Coordinating Center

The Medical Director leads the overall scientific and administrative direction of CINRG. Specifically, the medical director focuses on identifying potential targets for pharmacological trials; initiates collaborations for the design of in vivo and in vitro pharmacological research in appropriate animal models of the diseases being studied; analyzes hit drugs; in collaboration with the CINRG therapeutic subcommittee members, evaluates possibilities for moving forward to human trials within the CINRG network; initiates new protocol development. The medical director directly interacts with the Scientific Review Committee, the Data Safety and Monitoring Board, the regulatory organizations and funding agencies to review and implement their recommendations.

Dr. Leshner was recruited from Children’s Hospital and the Medical College of Virginia in Richmond, where he directed the pediatric and adult MDA clinics for over twenty years. He was Professor of Neurology, Pediatrics and Physical Medicine and the recipient of the MCV "Clinician of the Year Award." He is currently serving as Medical Director of the CINRG network and as a liaison with biotech and pharmaceutical industries. He is responsible for oversight of recruitment progress for currently funded studies and for orchestrating the recruitment of international CINRG site patients into the natural history study. Bob is the proud father of 5 nearly self supporting children and a doting grandfather to 3 hyperkinetic and adorable kids.

The Scientific Director is responsible for the overall direction of the research program. He/She works closely with scientists to determine the potential of future studies ensuring that CINRG supports the most promising research endeavors. Additionally, he/she continually evaluates funded research and works to form partnerships to greatly expand the resources available to CINRG. Finally, he/she serves as a spokesperson and advocate for CINRG, representing CINRG at scientific meetings, advancing the support for research with policy-makers, donors, the media, and the broader science community.

Eric Hoffman
Dr. Hoffman has been involved in translational human molecular genetics, and applications of genomics to health problems since the mid 1980’s. He has over 300 publications, and is among the most highly cited researchers. Research milestones include identification of dystrophin, defining cause of first voltage sensitive ion channel disorders in humans and horses, molecular basis of recurrent pregnancy loss, and treatment of large animal models of Duchenne dystrophy with exon-skipping therapy. His research facility in Washington DC has integrated state-of-the-art basic genetics research (genome, transcriptome, and proteome), an international clinical trial network, and ethics research. Current research efforts include therapeutics of muscular dystrophy, exercise studies, diabetes research, and medical rehabilitation. The Center houses six federally funded genomics and proteomic cores.

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The Director of the Statistics and Data Management Center (SDMC) of CINRG oversees and provides leadership for all the activities involved in implementing CINRG clinical research studies at the sites. The SDMC supports the protocol development process. Once a protocol is approved, the SDMC works with sites on their IRB approval, and site initiation. The SDMC Director oversees the development of the database and data capture processes to collect study data. The Director oversees the development of a study-specific Manual of Operations as well as CINRG-wide Standard Operation procedures. The SDMC oversees quality assurance, performs site monitoring visits for site performance and adherence to protocol, safety reporting, and provides regulatory oversight. The SDMC Director oversees all statistical analyses associated with reports, presentations and publications. The SDMC Director works closely with the Scientific and Medical Directors of CINRG as well as with the Executive Committee, Data and Safety Monitoring Board, and Scientific Advisory Committee for CINRG. On a day-to-day basis the SDMC director works with CINRG’s project manager(s), central clinical evaluator(s), data manager(s), statistician(s), and regulatory staff.

Avital Cnaan, PhD, joined the Children’s National Medical Center (CNMC) in May, 2008 as Director, Multi-Center Studies Section in CNMC’s Children’s Research Institute. Prior to joining CINRG she was Professor of Biostatistics in Pediatrics at the University of Pennsylvania School of Medicine, as well as Chief of the Division of Biostatistics and Epidemiology at the Children’s Hospital of Philadelphia (CHOP). She continues to hold an adjunct professor appointment at the University of Pennsylvania. Dr. Cnaan has over 20 years experience in clinical research and multi-center clinical trials. She is the PI of the Coordinating Center for The Childhood Absence Epilepsy Study. As a statistician, her interests are in survival analysis, longitudinal analysis methods, and issues in design and analysis of clinical trials, such as compliance and site effects. She is an author on more than 120 peer-reviewed publications and a Fellow of the American Statistical Association.

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The Project Manager, PM, is responsible for the overall project management of clinical studies including management of all clinical research associates and management of contractors needed to run clinical trials (central laboratories, electronic data capturing system, etc). The PM works with senior management to define project’s corporate strategic direction. They schedule, facilitate, chair, and document project team meetings; lead project team to develop project implementation strategy, milestones, and timelines that align with corporate strategy. Together with senior management, they develop project budgets and manage project expenses against approved budget. They facilitate information flow between project teams and senior management; and motivate and evaluate performance of project team members. They also coordinate and ensure completion of scientific documents including protocols, clinical study reports, investigator brochures and other regulatory or study related documents.

Lauren Hache graduated from the University of Miami in 2003 with a Bachelor’s Degree in Microbiology and Immunology. She has been working in the field of clinical research for the past eight years. Ms. Hache has 4 years of experience as a program manager for multi-site DMD clinical studies and on the leadership team of CINRG. This has involved extensive regulatory, site monitoring, trouble-shooting, budgetary, and other experience. Ms. Hache expects to obtain a masters degree in genetic counseling from the Graduate School of Public Health at the University of Pittsburgh in 2009. Dr. Clemens serves as her mentor.

Angela Zimmerman graduated in 1994 from the University of Iowa and completed a M.S. in Clinical Trial Administration from George Washington University in 2008. She joined CINRG in 2004 and has been instrumental in operational execution of clinical trials. She has over ten years of experience conducting multiple clinical trials with sites worldwide and has a strong track record in operations management, including management of multi-side clinical trials, study budgets, and protocol timelines.

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The Data Manager is responsible for the overall quality of data collected by CINRG centers. She helps design the case report forms and continually checks the completeness and accuracy of the data (including working with the Clinical Evaluator Trainer on the data from the CINRG Quantitative Muscle Testing (CQMT) system). She develops and maintains data testing methodology, and works with the Project Manager to identify and improve performance in the overall study data management process. The Data Manager oversees production of data reports (including graphs and tables) to the Data and Safety Monitoring Board and for manuscript publication. She also works with the Operations Director on supervising contractors in the development of data collection materials (including case report forms).

Adrienne Arrieta completed her Master’s Degree in Biostatistics in 2004 from George Washington University. She was previously working on large multicenter clinical trials in the Maternal Fetal Medicine Unit at George Washington University’s Biostatistics Center. There she was in charge of the accuracy and completeness of the data as well as communicating with the nurses on study procedures.

Fengming Hu completed her Master’s Degree in Statistics in 2005 from Virginia Tech. She was previously working in Department of Biostatistics at Georgetown University as volunteer biostatistician and many projects of data analysis in medical field, including DMD clinical trial data analysis. After she joined CINRG, she was in charge of the accuracy and completeness of the data for CINRG study and did interim and final data analysis as a biostatistician.

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The Clinical Evaluations (CE) Manager is involved in preparing new clinical research centers to begin testing as part of CINRG and in maintaining standard testing procedures and practices within and between the sites. This includes coordinating the set up of the site test facilities, managing equipment/supplies for CQMS, training and recertifiying clinical evaluators, monitoring quality and consistency of clinical evaluations outcome data, assisting in CQMS-related protocol development, as well as providing ongoing support to the clinical testing center and evaluators. The CE manager collaborates with other CINRG subcommittees and neuromuscular networks for outcomes development to better gather and share meaningful information to facilitate progress in neuromuscular research.

Tina Duong is a research physical therapist who manages and coordinates the standardization and training for clinical evaluations of the network. She graduated from the University of Oklahoma with a Masters of Physical Therapy. Her research interests lie in the area of strength and outcome measures, with expertise in the areas of pediatrics and neuro-rehabilitation. In addition, she is a co-investigator in a preclinical study focusing on exercise and strength in different mouse strains with muscular dystrophy, as well as a clinical trial assessing strength measures in children with DMD. She is also the physical therapist liaison for the MDA clinics in Washington DC and Virginia.

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A Study Chair is the primary investigator in charge of a specific project. They provide the overall direction and leadership for the study, and they are the main link between the other participating investigators around the world. The study chair leads a team of researchers to define a topic of interest and to formulate a question for study and to decide what techniques will be best for answering that question. Once a study is developed, the Study Chair leads the team of investigators to conduct and complete the study. He or she is responsible for all aspects of the study, including participant recruitment, making sure that participants are all treated according to the study protocol, that participant safety is being monitored, that information about the study is collected appropriately, and that the study funding is managed well. Once a project is complete, the Study Chair leads the group of investigators to analyze the data and write study reports or articles in journals so that they can share their study results with the scientific, medical and patient communities.

Erik Henricson is a co-founder of CINRG, the Co-Principal Investigator of the CDC-sponsored National Initiative for Families with Duchenne and the new DMD natural history study conducted by CINRG and the University of California at Davis. In addition to those studies, he has served as a co-investigator on several past and active CINRG clinical trials. He currently sits as a member of the Muscular Dystrophy Association’s Clinical Research Network Steering Committee. He received his Master’s degree in Public Health from the George Washington University in 1999, and a BS in Health Policy from the Pennsylvania State University in 1993.

Dr. Clemens is Associate Professor of Neurology at the University of Pittsburgh and chief of the neurology service at the Pittsburgh VA Healthcare System. Her clinical subspecialty is neuromuscular disease, principally focused on adult patients. She is a site investigator for CINRG and the Principal Investigator on a CINRG-sponsored trial investigating potential therapeutic agents in non-ambulatory DMD patients. She also heads a basic research laboratory focused on improving gene transfer to skeletal muscle.

Dr. Craig M. McDonald is a professor in Physical Medicine and Rehabilitation and Pediatrics at the University of California, Davis and the Medical Director Rehabilitation, Shriners Hospital for Children Northern California. Dr. McDonald is the director of the Rehabilitation Research and Training Center in Neuromuscular and a principal investigator on the CINRG study UCD0305, "A Longitudinal study of the relationship between impairment, activity limitation, participation and quality of life in persons with confirmed Duchenne muscular dystrophy (DMD)." Areas of research interest include management of childhood progressive neuromuscular disease, energy expenditure during locomotion, qualitative measurement of physical activity, pediatric trauma spina bifida, and managing spasticity in children with cerebral palsy. Dr. McDonald received the Young Academician Award, 1997 presented by the Association of Academic Physiatrists for outstanding performance in teaching, research and academic administration.

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