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

NCT ID: NCT07041203

Last Updated: 2025-11-26

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-03-01
• Study Completion Date: 2027-07-31

Brief Summary

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.

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. 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.

Conditions

Spasmodic Dysphonia; Laryngeal Dystonia; Voice Tremor

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Clinical response to sodium oxybate (Lumryz)

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

Group Type: EXPERIMENTAL

sodium oxybate

Intervention Type: DRUG

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

Interventions

sodium oxybate

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

Intervention Type: DRUG

Other Intervention Names

Lumryz

Eligibility Criteria

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

• Minimum Eligible Age: 21 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Kristina Simonyan

OTHER

Sponsor Role: lead

Responsible Party

Kristina Simonyan

Professor of Otolaryngology-Head and Neck Surgery

Responsibility Role: SPONSOR_INVESTIGATOR

Locations

Massachusetts Eye and Ear

Boston, Massachusetts, United States

Countries

United States

Central Contacts

Kristina Simonyan, MD, PhD, DrMed

Role: CONTACT

kristina_simonyan@meei.harvard.edu

6175736025

Facility Contacts

Kristina Simonyan, MD, PhD, DrMed

Role: primary

kristina_simonyan@meei.harvard.edu

6175736016

Other Identifiers

2025P000919

Identifier Type: -

Identifier Source: org_study_id