NCT03292458

Sodium Oxybate in Spasmodic Dysphonia and Voice Tremor

Central Mechanisms and Treatment Response of Sodium Oxybate in Spasmodic Dysphonia and Voice Tremor

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 117 (actual)
• Sponsor: Kristina Simonyan · Academic / Other
• Sex: All
• Age: 21 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

Using a comprehensive approach of clinico-behavioral testing, neuroimaging and pharmacogenetics, the researchers will examine the clinical effects of sodium oxybate and the matched placebo on voice symptoms in spasmodic dysphonia and voice tremor.

Detailed description

Spasmodic dysphonia (SD), or laryngeal dystonia, is a chronic debilitating condition that selectively affects speech production due to involuntary spasms in the laryngeal muscles. SD often extends beyond the impairment of vocal communication causing significant occupational disability and life-long social isolation. SD becomes even more incapacitating when it is associated with dystonic voice tremor (VT), which is present in about 1/3 of SD patients and is characterized by the inability to sustain a vowel for more than a few seconds. Current treatment of these disorders is limited to the temporary management of voice symptoms with repeated injections of botulinum toxin into the laryngeal muscles. These injections, however, are not fully effective in all SD patients and even less so in combined SD and VT cases. There is, therefore, a critical need to identify alternative therapeutic options that specifically target the pathophysiology of these disorders. On the other hand, the design and the use of such novel therapeutic approaches will be largely unattainable if their central mechanisms of action remain unknown. The objective of this study is to elucidate the primary determinants of clinical response to a novel oral medication, sodium oxybate (Xyrem®), in alcohol-responsive SD and VT patients. Using a comprehensive approach of clinico-behavioral testing, neuroimaging and pharmacogenetics, we aim to determine the clinical response of SD and VT symptoms to sodium oxybate and identify the primary markers of its clinical benefits. This study will use a controlled experimental design that focuses on detailed characterization of primary effects of a novel oral medication, sodium oxybate, for treatment of SD and VT symptoms.

Conditions

• Spasmodic Dysphonia
• Voice Tremor

Interventions

Type	Name	Description
DRUG	Sodium Oxybate	Alcohol challenge test and oral administration of a single dose of sodium oxybate and the matching placebo.

Timeline

• Start date: 2018-01-22
• Primary completion: 2021-12-29
• Completion: 2024-10-11
• First posted: 2017-09-25
• Last updated: 2025-12-30
• Results posted: 2025-04-04

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study