Current Clinical Trials

Delineating Physiologic Mechanisms of Swallowing impairment and Decline in Amyotrophic Lateral Sclerosis
R01 NS100859-01 National Institute of Neurological Disorders and Stroke

Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease causing progressive muscle weakness and impairments in swallowing (dysphagia) leading to malnutrition, dehydration, aspiration pneumonia, and ultimately death. The purpose of this research study is to understand the natural appearance and progression of swallowing impairment throughout disease progression and to identify a set of sensitive, easy to administer clinical tests that can accurately detect swallowing impairment for easy implementation into ALS clinics. This work will produce a highly practical and conceptually novel dysphagia risk index tool (PRISIM). 110 individuals with a diagnosis of ALS will complete a comprehensive evaluation of speech, breathing, swallowing and cough function every three months in the ARC laboratory at the University of Florida. Information derived from this study will produce the first natural history model of bulbar decline for swallowing function in ALS. This study is funded by the National Institutes of Health Division of Neurologic Disorders and Strokes (NINDS). 

Physiological Flow of Liquids Used in Dysphagia Management
R01 DC011020 National Institute of Deafness and other Communication Disorders

The practice of thickening liquids has become one of the most frequently used interventions for swallowing impairment (dysphagia). Surprisingly however, it is not yet established how this intervention impacts swallowing physiology and bolus flow. This study is directed by Dr. Catriona Steele for the Swallowing Rehabilitation Research Laboratory at the Toronto Rehabilitation Institute and will examine the impact of liquid rheology or viscosity on bolus physiological flow during swallowing. The ARC laboratory will specifically focus on swallowing outcomes in neurodegenerative diseases and will include individuals with ALS (enrollment completed) and Parkinson’s disease (currently enrolling). Enrolled participants will attend the ARC laboratory at the University of Florida one time and undergo a videofluoroscopic swallowing evaluation. This study is funded by the National Institute on Deafness and Other Communication Disorder.  

Molecular Characterization of ALS/FTD in a Novel C9ORF72 BAC Mouse Model
R01 NS098819 National Institute of Neurological Disorders and Stroke

This basic science study lead by ARC collaborator, Dr. Laura Ranum in the Center for Neurogenetics and will investigate molecular mechanisms underlying the most common familial form of ALS. She was one of the pioneers in the field to discover that this form of genetic ALS is caused by the expansion of a microsatellite GGGGCC repeat in the C9orf72 gene. Her lab generated a BAC transgenic model of C9-ALS/FTD that will be used in this study to define pathogenic mechanisms underlying C9-ALS/FTD disease development and progression and provide an accessible and well-characterized mouse model for therapeutic development. Dr. Plowman is serving as a Co-Investigator and will lead a series of experiments assessing bulbar dysfunction in the transgenic ALS mouse model.

Lung Volume Recruitment Therapy with Expiratory Muscle Strength Training in ALS
ALS Association Clinical Management Grant

Dysphagia, dystussia (cough impairment), and respiratory impairment are hallmark features of ALS. Recent evidence by Dr. Plowman suggests that mild to moderate intensity respiratory exercises applied early in the disease progression can improve and maintain these vital functions. The study is being directed in conjunction with Co-Principal Investigator David Walk, and the purpose of this study is to investigate the effects of two treatment techniques, Expiratory Muscle Strength Training (EMST) and Lung Volume Recruitment (LVR), on breathing, swallowing, speech, and cough function in early affected individuals with ALS. Previous work by Dr. Plowman indicated EMST to be an effective treatment method for improving cough function and swallowing in ALS. LVR is a technique performed with a resuscitation bag fitted with a mouthpiece and a one-way valve to increase cough function and passive expansion of the lungs. In this randomized control trial in partnership with the University of Minnesota, participants are assigned to one of two treatment groups- EMST intervention alone or combined treatment of EMST and LVR. This study is funded by the Amyotrophic Lateral Sclerosis Association. 

Impact of Nuedexta on Swallowing, Speech and Cough Physiology in ALS
ALS Association Clinical Management Grant

Speech and swallowing difficulties occur throughout disease progression in individuals with ALS who rate this symptoms as the worst aspect of the disease (Hillel and Miller, 1989).  These impairments lead to social isolation and withdrawal, reduced quality of life and mental wellbeing, malnutrition, aspiration pneumonia and death. A current gap in the clinical care of individuals with ALS is effective treatments to either improve or maintain speech and swallowing function. An exciting and promising drug that is already approved by the FDA for use in ALS patients, Nuedexta, was recently documented to improve patient-rated speech and swallowing function (Smith et al., 2016). While this is very promising, no study has yet determinte the impact this pharmacologic intervention has on objective clinical and physiologic outcomes of speech and swallowing in ALS patients. The primary goal of this proposal is to rigorously evaluate the impact of a pharmacological treatment, Nuedexta, on swallowing physiology, airway protection and speech intelligibility in individuals with ALS. Outcomes of this work will provide valuable physiologic insight into a promising therapeutic and help to reduce current gaps in the clinical management of individuals with ALS.

Dr Emily Plowman and ARC Alum Dr. Lauren Tabor-Gray of the Phil Smith Neuroscience Institute in Fort Lauderdale will be recruiting 30 individuals with ALS who also demonstrate impairments in bulbar function and who have not yet taken Nuedexta. Participants will be studied before and immediately after a 30 day course of the drug to compare physiologic outcomes of bulbar function.

Examining the therapeutic potential of an FDA-approved drug Metformin for C9orf72 ALS/FTD
UF Moonshot PIlot Grant

Metformin is an FDA approved drug used with a diet and exercise to control high blood sugar in adults with type II diabetes. Preclinical studies conducted by Dr. Laura Ranum at the University of Florida demonstrate that Metformin may improve C9orf72 ALS symptoms in a transgenic mouse model. This study aims to determine if Metformin is a safe and potentially viable therapeutic treatment for individuals with C9orf72 ALS. This open-label study is funded by the Center for NeuroGenetics in the College of Medicine at the University of Florida and the University of Florida Moonshot Neuromuscular Health Initiative. We are recruiting individuals with a confirmed C9orf72 ALS diagnosis who will be studied over a one year period.

Improving Detection and Clinical Care of Dysphagia in Cardiac Surgical Patients
UF Department of Surgery Pilot Grant

Dysphagia is a common complication following cardiac surgery leading to delayed return to oral intake, increased risk of dehydration and malnutrition, intubation, and death. Dysphagia- linked aspiration pneumonia is a potentially preventable condition and is the leading cause of mortality following open heart surgery. While dysphagia is a known complication of cardiac procedures, little is known regarding potential demographic, clinical, or surgical factors that place a patient at higher risk for developing dysphagia. In addition, our preliminary data shows that nurses lack sufficient training in performing swallowing screens and lack sensitive screening tools to detect dysphagia. Therefore, this study aims to (1) identify risk factors for postoperative dysphagia and create a clinically useful online dysphagia risk stratification tool (CRISM-C), (2) identify a set of sensitive and easy to administer bedside markers of dysphagia to develop and validate a nursing screening tool, and (3) development of an educational nursing learning platform. This study is being conducted with ARC collaborator Dr. Eric Jeng and is sponsored by the Departments of: Speech, Language and Hearing Science and Thoracic and Cardiovascular Surgery at University of Florida. 

Past Projects:

Upper Airway Sensory Degradation in Individuals with ALS
Clinical and Translational Science Institute (CTSI) Pilot Grant

Amyotrophic Lateral Sclerosis Association Clinical Management Grant
Impact of Respiratory Training in Amyotrophic Lateral Sclerosis

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National Institute of Child Health Development
R21 HDO75327 Effects of Strength Training on Bulbar Function in Persons with Amyotrophic Lateral Sclerosis

National Institute of Deafness and Other Communication Disorders
R03 DC012123 Dissociating the Neural Substrates of Cranial & Limb Motor Impairment in an Animal Model of PD

National Institute of Deafness and other Communication Disorders
F32 DC010569 Ruth L. Kirschstein National Research Service Award F32 Individual Post-Doctoral Fellowship
Neural Substrates of Oral Motor Dysfunction in Parkinson’s Disease

Veterans Affair Office of Rural Health
N08-FY14Q4-S0-P01503 Reducing Barriers to living at home for the Rural Veteran with Amyotrophic Lateral Sclerosis

University of South Florida College of Medicine Interdisciplinary Seed Grant
IS415-3 Impact of Lingual Resistance Exercise Training on Swallowing Function, Airway Protection and Oral Intake in Neurodegenerative Disease
Principal Investigator

University of South Florida Research and Innovation Grant
Impact of Exercise on Disease Progression and Survival in a Transgenic Mouse Model of ALS
Principal Investigator

Joy McCann Center for Swallowing Disorders Research Foundation
Impact of EMST on Bulbar Function in Amyotrophic Lateral Sclerosis: A Pilot Study
Principal Investigator

Ruth L. Kirschstein National Research Service Award T32 Institutional Training Fellowship
National Institute of Deafness and other Communication Disorders