Extended-release Sodium Oxybate (Lumryz) in Spasmodic Dysphonia and Voice Tremor

Phase 1Not Yet RecruitingNCT07041203

Kristina Simonyan8 enrolled

Overview

Using a comprehensive approach of clinico-behavioral testing and neuroimaging, the researchers will examine the clinical effects of the extended-release formulation of sodium oxybate on voice symptoms in spasmodic dysphonia in an open-label, proof-of-concept, dose-finding study.

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. Treatment of SD is limited to injections of botulinum toxin into the vocal cords, however, it is often only partially effective and can have side effects. More than half of the people with SD have some relief from drinking alcohol. The previous studies showed that immediate-release sodium oxybate (an oral drug that acts similarly to alcohol) significantly relieves voice symptoms in patients with alcohol-responsive SD. In this study, we will examine the efficacy and safety of extended-release sodium oxybate formulation (Lumryz) as a longer-acting oral agent for the treatment of patients with alcohol-responsive SD.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Spasmodic Dysphonia Laryngeal Dystonia Voice Tremor

Interventions

sodium oxybateDRUG

Oral administration of sodium oxybate (1.5g, 2.0g, 2.5g, 3.0g)

Eligibility

Sex: ALLMin age: 21 YearsMax age: 80 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Provision of signed and dated informed consent form 2. Stated willingness to comply with all study procedures and lifestyle considerations (see Lifestyle Considerations below) and availability for the duration of the study 3. Males and females 4. Age 21-80 years 5. Documented diagnosis of alcohol-responsive laryngeal dystonia 6. Documented positive response to immediate-release sodium oxybate (Xyrem) in prior studies 7. Willingness to adhere to the study intervention regimen Exclusion Criteria: 1. The incapability of giving informed consent 2. Pregnancy or breastfeeding until a time when they are no longer pregnant or breastfeeding 3. Grade 2 or higher hepatic or renal dysfunction according to the NCI criteria 4. Moderate to severe congestive heart failure 5. Cognitive impairment (MoCA \< 26) 6. Past or present suicidal ideations (according to C-SSRS) 7. Alcoholism or high risk for alcohol use disorder according to the NIAAA definition and DSM-5 criteria 8. Asymptomatic presentation due to botulinum toxin treatment until the time they are fully symptomatic and are at least 3 months after the last injection 9. Increased daytime sleepiness (Epworth Sleepiness Scale (ESS\>10)) 10. Past or present history of any neurological disorders (except for LD and co-occurring voice tremor), such as stroke, movement disorders, brain tumors, traumatic brain injury with loss of consciousness, ataxias, myopathies, myasthenia gravis, demyelinating diseases, alcoholism, drug dependence. 11. Past or present history of any psychiatric problems, such as schizophrenia, major and/or bipolar depression, or obsessive-compulsive disorder 12. Current use of medication(s) affecting the central nervous system 13. Past or present history of brain and/or laryngeal surgery 14. Presence of certain tattoos, ferromagnetic objects in their bodies (e.g., implanted stimulators, surgical clips, prosthesis, artificial heart valve, etc.) that are not MRI comparable and/or cannot be removed for the purpose of MRI study participation

Locations (1)

Massachusetts Eye and Ear

Boston, Massachusetts, United States

Outcomes

Primary Outcomes

Efficacy of Lumryz

The primary outcome will be the percent change from baseline in LD symptoms 60 min after each dose of drug intake

Time frame: Through study completion, an average of 4 days

Efficacy of Lumryz

The primary outcome will be the number of patients (N, % of total) with improved voice symptoms from baseline after each dose of the drug.

Time frame: Through study completion, an average of 4 days

Secondary Outcomes

Duration of treatment efficacy

The secondary outcomes will be the duration (in hours) of Lumryz treatment efficacy

Time frame: Through study completion, an average of 4 days

Central Contacts

Kristina Simonyan, MD, PhD, DrMed

CONTACT

6175736025 kristina_simonyan@meei.harvard.edu

Data from ClinicalTrials.gov

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