WORKSHOP ON CLINICAL TRIAL DESIGN FOR
CARDIORESPIRATORY DYSFUNCTION
MARCH 22 - 23, 2010
The Clinical Trial Design Workshop, sponsored by the Rett Syndrome Research Trust, was organized as a proactive approach to addressing the symptoms of autonomic dysfunction in Rett Syndrome which cause distress, compromise overall health and are in some cases life-threatening. Our goal was to develop ideas and consensus to create an immediate plan of action, defining and confronting these problems in a context of evidence-based future treatments.
The workshop was held in the form of a round table discussion with the following participants:
- Basic scientists with experience and success in the evaluation and treatment of respiratory dysfunction in animal models.
- Multidisciplinary physician-scientists with experience in treatment of patients with Rett Syndrome.
- Clinicians and researchers not familiar with Rett Syndrome who are leading experts in the treatment of similar symptoms in patients with other diseases; research related to the central regulation of cardiorespiratory functions and design of clinical research trials.
Unlike conventional meetings, where scientists or clinicians discuss topic of interest among themselves, this meeting was organized specifically to facilitate the cross-pollination of ideas between these various specialties. This interdisciplinary format promoted valuable sharing of both experiences and unpublished work. The feeling of responsibility toward those suffering with Rett Syndrome strongly permeated all the discussions. There was an almost palpable sense of having a real patient in the center of the room where words and thoughts of this competent group intersected.
Topics were wide-ranging and included subjects such as the medical consequences of low CO on cerebral blood flow; kidney function; tachycardia that follows breath holds. Differences between the effects of certain drugs in mice and in humans were delineated and risk factors defined. This work is essential to the sound construction of drug trials, two of which are now being designed at Montefiore.
A report on the workshop will be presented on April 18th at the Montefiore Parent gathering and subsequently posted on line.
Aleksandra Djukic Ph.D., M.D.
Director Rett Syndrome Center
Children's Hospital at Montefiore
PARTICIPANTS:
Bissonnette John, M.D.
Professor of Obstetrics and Gynecology and of Cell and Developmental Biology
Oregon Health & Science University (Portland, OR)
Ben Zeev Bruria, M.D.
Associate Professor of Pediatrics
Head of Pediatric Neurology Unit and Israeli Rett Center
Safra Pediatric Hospital, Sheba Med Center (Ramat-Gan, Israel)
Djukic Aleksandra M.D., PhD
Associate Professor of Neurology
Director, Rett Syndrome Center
Montefiore Medical Center
Albert Einstein College of Medicine (Bronx, NY)
Dutschmann Mathias, PhD
Senior Lecturer in Physiology Institute of Membrane and Systems Biology, University of Leeds, UK (Leeds, UK)
Erickson Craig, M.D.
Assistant Professor of Psychiatry,Indiana University School of Medicine, and the Christian Sarkine Autism Treatment Center, James Whitcomb Riley Hospital for Children (Indianapolis, IN)
Galanopoulou Aristea, M.D., PhD
Associate Professor of Neurology and Neuroscience
Albert Einstein College of Medicine (Bronx, NY)
Gates Gregory, PhD
Cardiopulmonary Physiology
Autonomic function monitoring
Rett Syndrome Center at Montefiore
Albert Einstein College of Medicine (Bronx, NY)
Guyenet Patrice, PhD
Professor of Pharmacology
University of Virginia (Charlottesville, VA)
Katz David, PhD
Professor, Neuroscience,
Case Western University (Cleveland, OH)
Moshe Solomon M.D.
Professor of Neurology, Neuroscience, and Pediatrics
Vice chairman of the Department of Neurology
Director of Child Neurology and Clinical Neurophysiology
Martin A. and Emily L. Fisher fellowship in Neurology and Pediatrics
Albert Einstein College of Medicine (Bronx, NY)
Muzumdar Hiren, MD
Assistant Professor of Pediatrics (Pulmology)
Rett Syndrome Center
Albert Einstein College of Medicine (Bronx, NY)
Neul, Jeffrey, M.D., Ph.D.
Assistant Professor, Section of Neurology, Department of Pediatrics
Assistant Medical Director, Blue Bird Circle Rett Clinic
Baylor College of Medicine (Houston, TX)
Ramirez Jean Marino PhD
Professor, Neurological Surgery
Director, Center for Neuroscience
Seattle Children's Research Institute (Seattle, WA)
Robertson David M.D.
Professor of Medicine, Pharmacology and Neurology
Elton Yates Professor of Autonomic Disorders
Director of the Center for Space Physiology and Medicine
Vanderbilt University (Nashville, TN)
Sinnett Mark, PHARM D, FASHP
Director, Clinical and Educational Services
Department of Pharmacy
Montefiore Medical Center
Albert Einstein College of Medicine (Bronx, NY)
Shinnar Shlomo M.D., PhD
Professor of Neurology, Pediatrics and Epidemiology and Population Health
Hyman Climenko Professor of Neuroscience Research
Director, Comprehensive Epilepsy Management Center,
Montefiore Medical Center, Albert Einstein College of Medicine (Bronx, NY)
Walsh Christine, M.D. FAAP, FACC
Professor of Clinical Pediatrics
Divisions of Pediatric Cardiology and Critical Care Medicine
Director, Pediatric Dysrhythmia Center
Co-Director, Montefiore Einstein Cardio-Genetics Center
Cardiologist, Rett Syndrome Center at Montefiore (Bronx, NY)