Governance

The CINRG network is governed by a by-law document to ensure its' research and clinical activities follow principles of open and full scientific communication, peer review, full disclosure of potential conflicts of interest, and democratic principles. An elected Executive Committee provides guidance and direction for the network and is advised by a Scientific Advisory Committee. The Scientific Advisory Committee (SAC) is composed of independent physicians and scientists from academia, non-profit organizations/foundations and the government with expertise in Neuromuscular Disorders and is appointed by CINRG's Scientific Director. The SAC recommends research priorities and reviews the network's progress on a rolling basis. A Data Safety Monitoring Board, convened at least annually by the Coordinating Center, reviews all active protocols and ensures that all studies are conducted safely and adhere to federal guidelines for safety standards. The seven member team has extensive experience in clinical research and safety monitoring in a variety of disease states, including neuromuscular disorders and includes patient representatives.

Executive Committee

CINRG is led by an elected Executive Committee composed of some of the leading researchers in Neuromuscular Disorders plus some of the Principal Investigators from the CINRG Network. This combination of NMD scientific knowledge combined with an in depth understanding of CINRG provides excellent leadership for CINRG. The Executive Committee Members are listed below.

Medical Director: Paula Clemens, MD
Dr. Clemens' activities include patient care, with a specific emphasis on inherited myopathies, clinical research in Duchenne Muscular Dystrophy and late-onset Pompe disease and basic laboratory research. She directs the neurology service of the Pittsburgh PA Healthcare System. She is actively involved in both graduate and medical education.

Dr. Clemens directs an active basic research laboratory focused on gene transfer for the treatment of skeletal muscle diseases. The principal disease models studied in her laboratory are Duchenne muscular dystrophy and muscle cachexia. Her laboratory pursues studies of in utero gene transfer to muscle using both intramuscular and systemic delivery approaches. Her laboratory has a long-standing interest in the immunology of gene transfer for muscle disease. The current direction of this research is to characterize the role of regulatory T cells in the muscle pathology of the mouse model of Duchenne Muscular Dystrophy, with particular emphasis on the setting of therapeutic dystrophin gene transfer.

Dr. Clemens is the study chair for two CINRG studies: PITT0908 Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophy and PITT1109 Cardiac Outcome Measures in Children with Muscular Dystrophy. Dr. Clemens has been a member of the CINRG Executive Committee since its inception in 2008. She is also the Chair of the CINRG Publication Subcommittee.

Scientific Director: Eric Hoffman, PhD
Eric Hoffman, PhD earned his PhD in Genetics at Johns Hopkins University and then pursued post-doctoral training with Louis Kunkel at Boston Children's Hospital working on Duchenne Muscular Dystrophy gene and protein identification. He is the director of the Research Center for Genetic Medicine, and James Clark Professor of Pediatrics at Children's National Medical Center in Washington, DC. The center has integrated state-of-the-art basic genetics research (genome, transcriptome, and proteome), with an international clinical trial network, and ethics research. He has a diverse portfolio of research, and his Center hosts national research cores for rehabilitation medicine, muscular dystrophy, developmental disorders, and clinical and translational research. He has over 400 publications. Hoffman's goals are to conduct research on inherited conditions, with emphasis on gene identification (positional cloning and candidate gene analyses), pathophysiological studies, molecular diagnostics, and therapeutics.

Dr. Hoffman founded CINRG in 1999 and has provided continuity and leadership to CINRG since then. He has been the Scientific Director for CINRG since 2008.

Professor Hoffman is board-certified by the American Board of Medical Genetics in Clinical Molecular Genetics.

Coordinating Center Director: Avital Cnaan, PhD
Avital Cnaan, Ph.D. has more than 20 years experience as a statistician in multi-center clinical research studies. For the past 15 years, Dr. Cnaan has been directing coordinating centers of federally funded multi-center clinical research studies. Prior to joining CNMC she was Professor of Biostatistics in Pediatrics at the University of Pennsylvania School of Medicine, as well as founder and Chief of the Division of Biostatistics and Epidemiology at The Children's Hospital of Philadelphia (CHOP). Her diverse experience, including fifteen years on CHOP's Institutional Review Board (IRB), provides a broad appreciation for the complexities and ethical and regulatory issues in pediatric clinical research and strong experience in the process of protocol development, review, and implementation. Dr. Cnaan is a Professor of Pediatrics, Epidemiology and Biostatistics in the Schools of Medicine and Health Sciences and Public Health and Health Services at George Washington University. Dr. Cnaan is a Fellow of the American Statistical Association.

Washington University: Julaine Florence, DPT
Dr. Florence is Research Professor of Neurology and Director of Clinical Studies, Neuromuscular Division, Department of Neurology, Washington University School of Medicine, St. Louis, Mo, USA. The focus of her career has been on the design, implementation and optimization of clinical outcome measures for therapeutic trials in individuals with Neuromuscular Disorders. Julaine is also Professor of Physical Therapy, Program in Physical Therapy, Washington University School of Medicine and has been involved in rehabilitation services in the Neuromuscular Disease Clinic at Washington University, St. Louis since its' inception.

Carolinas Medical Center, Susan Sparks, MD
Susan Sparks, MD, PhD is a board certified Pediatrician with additional board certifications in Clinical Genetics and Clinical Biochemical Genetics. Dr. Sparks joined the Department of Pediatrics in the Division of Clinical Genetics at Levine Children's Hospital in March 2009. Before joining Levine's Children's Hospital, she was at Children's National Medical Center in Washington, DC. Dr. Sparks participates in general genetics, metabolic genetics, and Down's Syndrome clinics, in addition to inpatient consults in genetics and metabolism at Levine Children's Hospital and Carolinas Medical Center. In addition to general and metabolic genetics expertise, she has a particular interest in neuromuscular disorders with a biochemical basis.

University of California, Davis: Erik Henricson, MPH
Erik Henricson is a co-director of the NIDRR / UC Davis Rehabilitation Research and Training Center in Neuromuscular Disease in the UC Davis Medical Center's Department of Physical Medicine and Rehabilitation. He received his Master's Degree in Public Health in 1999 from the George Washington University School of Medicine and Health Sciences. He is a founding member of the CINRG group and has developed and participated in many of the group's clinical trials, and is currently serving in his sixth year as co-principal investigator of the CINRG study "A Longitudinal Study of the Relationship between Impairment, Activity Limitation, Participation and Quality of Life in Persons with Confirmed Duchenne Muscular Dystrophy (DMD). His research interests include development of functional outcome measures for clinical trials, the relationship between functional abilities and quality of life, and neurobehavioral functions of individuals with neuromuscular disorders. He is currently undertaking his PhD in human development, with a focus on cognition, quality of life and physical function in childhood and adolescence in individuals with neuromuscular disorders.

Royal Children's Hospital, Melbourne, Australia: Monique Ryan, MD
Dr. Ryan is Head of the Neuromuscular Clinic and Neuromuscular Clinical Research Program at the Royal Children's Hospital in Melbourne Australia. She trained as a pediatric neurologist and completed a Neurophysiology fellowship at the Children's Hospital Boston and the Lahey Clinic, prior to returning to Australia in 2003. Since 2003 she has been a CINRG PI, initially at the Children's Hospital at Westmead in Sydney, and now in Melbourne. She has been site PI on a number of CINRG trials in addition to the Ataluren and Prosensa trials in DMD, and is also actively involved in natural history and therapeutics trials in the inherited neuropathies. She is also a member of the extended committee of the TREAT-NMD Therapeutic Advisory Committee.

Fundacion Favalaro, Buenos Aires, Argentina: Alberto Dubrovsky, MD
Alberto Dubrovsky, MD, is currently Chairman of the Department of Neurology, Director of Neuromuscular Disease Unit of the Neurosciences at Fundacion Favaloro. He also serves as Medical Director of the Argentina Muscular Dystrophy Association since 1983. He is Professor of Neurology at the University of Buenos Aires for 23 years. Also has been designated as Full Professor of Neurosciences at the Favaloro University in 2008. He trained as Neurologist in the field of Neuromuscular Diseases at NIH under the sponsorship of W King Engel. Dr. Dubrovsky is past president of the Argentine Neurological Society and is a member of the Executive Committee-Research Group of Neuromuscular Diseases from the World Federation of Neurology. He has been instrumental in developing the neuromuscular specialty in Argentina as well as neuromuscular centers for patient care and research in his country. He is actively involved in Neuromuscular Diseases and EMG in clinical and research areas and has a special interest in Muscular Dystrophies and related disorders. He has served as Principal Investigator at the CINRG site since joining the network in 2005.

The Children's Hospital at Westmead, Sydney, Australia: Kristy Rose, PT, PhD
Kristy Rose is a clinical and postdoctoral research physiotherapist based in Sydney, Australia at The Institute for Neuroscience and Muscle Research, The Children's Hospital at Westmead. Kristy has over 10 years of clinical and research expertise in childhood neuromuscular disease and has been CINRG Clinical Evaluator since 2004. Her primary research interest is in outcome measurement for childhood muscle disease and was awarded a PhD for her work in this field in 2010. Since the award of her PhD Kristy continues with her clinical and research roles and has recently become a member of GlaxoSmithKline's Master Physiotherapist Training Team.

Carolinas Medical Center, Charlotte, NC: Priscilla Russo, RN, BSN
Priscilla Russo has been involved in clinical research since 1999. She began her research career with the position of RN/Clinical Research Coordinator for multiple sclerosis at the Neuroscience and Spine Institute at Carolinas Medical Center. In 2002, she was promoted to MS Research Program Coordinator and for the next five years, Priscilla successfully managed over 50 phase II to phase IV clinical research trials. During her tenure as Research Program Coordinator, Priscilla also served as a clinical advisor for the National MS Society-Mid Atlantic Chapter and as a nursing consultant for several biotechnology and biopharmaceutical companies. In 2008, Priscilla was promoted to the Clinical Research Manager at the Department of Neurology, Carolinas Medical Center. Currently she manages all aspects of over 60 clinical research trials (Phase I – IV) for Multiple Sclerosis, Amyotrophic Lateral Sclerosis, Parkinson’s Disease/Movement Disorders, Epilepsy, Stroke and Muscular Dystrophy. As the Clinical Research Manager for the Department of Neurology, Priscilla manages all aspects of clinical research: ensures adherence to Good Clinical Practice guidelines, manages study budget processes, prepares internal SOP guidelines, reviews and monitors study deviations and SAE/AE, oversees recruitment and retention of study participants, provides leadership to a research team of 15, and more.

CINRG Coordinating Center

The CINRG Coordinating Center (CC) works to monitor and establish both network and protocol specific standards to ensure the quality and safety of clinical research protocols run through the network. Specifically, the Coordinating Center provides study design assistance at study start as well as help in protocol writing, case report form design, database design and regulatory and ethical review. Once a study is approved, the coordinating center trains the sites on implementing the study and approves the site to allow recruitment of participants when training goals are accomplished. This includes, in particular, training of clinical evaluators at the sites on the assessments of outcomes in these studies. In addition, the Coordinating Center develops protocol specific manual of procedures (MOP), pharmacy manuals of operations and its own plans for monitoring the study progress and the sites while it is ongoing. Data quality and completeness is regularly reviewed. The Coordinating Center provides periodic data summaries to the Data and Safety Monitoring Board and also has a mechanism of alert in every study should there be a serious adverse event. At the conclusion of the study, the Coordinating Center analyzes the data and all involved CINRG members participate in its interpretation. Study results are presented at national and international meetings and are published in scientific journals. A brief description of the roles of the Coordinating Center and selected bios of key personnel are listed below.

Coordinating Center Director: Avital Cnaan, PhD
See above.

Medical Director: Paula Clemens, MD
See above.

Scientific Director: Eric Hoffman, PhD
See above.

Clinical Evaluations (CE) Manager: Tina Duong, PT
The CE manager supports the CQMS system, clinical evaluators and management of CINRG outcome measures. This includes training and certification of all site evaluators, quality assurance of data from clinical evaluations, CQMS related protocol development, maintenance of adequate operations at clinical trial sites and working with the vendor(s) who develop the CQMS and its component parts.

Operations Manager: Lauren Hache
The Operations Manager (OM) supports all activities related to protocol development, production and amendments, preparation and maintenance regulatory documents, manages the Coordinating Center grant and the patient care costs of the clinical trial sites, trains the clinical trial sites on protocol procedures, conducts internal monitoring as well as site monitoring visits, performs safety surveillance and facilitates communications.

Database Manager: Adrienne Arrieta and Mohammad Ahmed
The Database Manager (DBM) designs and maintains CINRG databases, designs and produces case report forms, tracks patients and case report form collection and conducts quality assurance activities. This role supports web-based and electronic communication systems designed to facilitate communication and exchange of information within CINRG. In addition, the DBM runs quality assurance checks on the data, generates data queries from the system and resolves queries.

Biostatistician: Fengming Hu and Heather Gordish-Dressman, PhD
The Biostatistician is responsible for review and input to protocol designs and development of sample size considerations and statistical analysis plans. Once a study is approved, the biostatistician reviews case report form development and proposed database design to ensure that it is consistent with protocol and future analysis needs. The biostatistician prepares all data summary reports for the DSMB and other groups, performs data analyses on the protocols and participates in interpretation and dissemination of results through presentations and preparation of manuscripts.