Study Purpose:
Nuedexta is FDA approved for the treatment of pseudobulbar affect in ALS patients and anecdotal reports of improvements in speech, salivation or swallowing have been reported. However, no prospective study has been conducted to comprehensively examine and determine the physiologic impact of Nuedexta on both speech and swallowing physiology in a large group of ALS individuals. These data are needed in order to provide evidence-based guidance to the management of bulbar dysfunction in ALS.Study Status:
Not recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
dextromethorphan HBr and quinidine sulfate
Placebo:
No
Phase:
Phase 1/Phase 2
Study Chair(s)/Principal Investigator(s):
Lauren Tabor, PhD, Phil Smith Neuroscience Institute at Holy Cross Hospital, Emily Plowman, PhD, University of Florida
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
University of Florida
Gainesville, Florida, 32610 United States
Full Study Summary:
Although advances in the management of bulbar dysfunction in ALS have been disappointing, recent interest has surfaced regarding the therapeutic potential of a pharmaceutical agent, Nuedexta (dextromethorphan HBr and quinidine sulfate), for the treatment of bulbar symptomology in individuals with ALS. Although Nuedexta received approval from the Food and Drug Administration (FDA) to target symptoms of pseudobulbar affect (PBA) in ALS; anecdotal reports of improvements in speech, salivation or swallowing were reported from Neurologists treating ALS individuals who were administered Nuedexta. Subsequently, a Phase II clinical trial was conducted that reported improvements in speech, swallowing and salivation following 30-days of Nuedexta treatment. One serious limitation of this study, however, is the fact that the primary outcome employed was a perceptual patient-report scale (PRO) (Center for Neurological Study Bulbar Function Scale, CNS-BFS), with no objective physiologic outcomes to confirm actual change in bulbar physiology. The absence of any objective clinical physiologic outcomes is particularly important when examining effects of Nuedexta, given that it contains selective serotonin reuptake inhibitors (SSRIs), or serotonergic antidepressants, that can impact the regulation of emotional expression, feelings of wellbeing and modulation of depression (all known to impact the response an individual will provide on a PRO measure). Furthermore, findings based on PRO's must be validated with studies that utilize objective physiologic outcomes of speech and swallowing function. Great excitement exists regarding the potential impact of Nuedexta on bulbar function in ALS with many neurologists prescribing Nuedexta to treat these symptoms in ALS patients. To date, however; no data exists to examine and determine the physiologic impact of Nuedexta on speech or swallowing physiology. These data are needed in order to validate the initial patient-reported outcomes of the Phase II clinical trial and to provide evidence-based guidance to the management of bulbar dysfunction in ALS.
Study Sponsor:
University of Florida
Estimated Enrollment:
28
Estimated Study Start Date:
07 / 25 / 2019
Estimated Study Completion Date:
11 / 22 / 2021
Posting Last Modified Date:
03 / 08 / 2023
Date Study Added to neals.org:
03 / 21 / 2019
Minimum Age:
18 Years
Maximum Age:
90 Years
Can participants use Riluzole?
Yes
Inclusion Criteria:- Diagnosis of probable-definite ALS (El-Escorial Criterion);
- ALSFRS-R Bulbar subscale score <10
- Bamboo oral reading speaking rate <140 words per minute
- No allergies to barium sulfate.
Exclusion Criteria:
- Treatment for sialorrhea within the past 3 months that includes either Botox or radiation treatment
- Participation in another disease modifying study targeting bulbar or cough function
- Use of invasive mechanical ventilation/presence of tracheostomy
- Advanced frontotemporal dementia or significant cognitive dysfunction
- Nil per oral status for feeding (i.e., NPO, nothing by mouth)
- Previously prescribed Nuedexta. Additionally, if participants are taking Riluzole or other medications to control sialorrhea, they must be on a stable dose for at least 30 days prior to enrollment in the current study.
Phil Smith Neuroscience Institute at Holy Cross Hospital
Fort Lauderdale, Florida
33308
United States
University of Florida
Gainesville, Florida
32610
United States