NCT07041203

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.

Trial Health

63
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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
8

participants targeted

Target at below P25 for phase_1

Timeline
12mo left

Started Oct 2026

Geographic Reach
1 country

1 active site

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 4, 2025

Completed
23 days until next milestone

First Posted

Study publicly available on registry

June 27, 2025

Completed
1.3 years until next milestone

Study Start

First participant enrolled

October 1, 2026

Expected
12 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2027

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2027

Last Updated

April 28, 2026

Status Verified

April 1, 2026

Enrollment Period

12 months

First QC Date

June 4, 2025

Last Update Submit

April 25, 2026

Conditions

Spasmodic DysphoniaLaryngeal DystoniaVoice Tremor

Outcome Measures

Primary Outcomes (2)

  • Efficacy of Lumryz

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

    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.

    Through study completion, an average of 4 days

Secondary Outcomes (1)

  • Duration of treatment efficacy

    Through study completion, an average of 4 days

Study Arms (1)

Clinical response to sodium oxybate (Lumryz)

EXPERIMENTAL

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

Drug: sodium oxybate

Interventions

sodium oxybateDRUG

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

Also known as: Lumryz
Clinical response to sodium oxybate (Lumryz)

Eligibility Criteria

Age21 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Provision of signed and dated informed consent form
  • Stated willingness to comply with all study procedures and lifestyle considerations (see Lifestyle Considerations below) and availability for the duration of the study
  • Males and females
  • Age 21-80 years
  • Documented diagnosis of alcohol-responsive laryngeal dystonia
  • Documented positive response to immediate-release sodium oxybate (Xyrem) in prior studies
  • Willingness to adhere to the study intervention regimen

You may not qualify if:

  • The incapability of giving informed consent
  • Pregnancy or breastfeeding until a time when they are no longer pregnant or breastfeeding
  • Grade 2 or higher hepatic or renal dysfunction according to the NCI criteria
  • Moderate to severe congestive heart failure
  • Cognitive impairment (MoCA \< 26)
  • Past or present suicidal ideations (according to C-SSRS)
  • Alcoholism or high risk for alcohol use disorder according to the NIAAA definition and DSM-5 criteria
  • Asymptomatic presentation due to botulinum toxin treatment until the time they are fully symptomatic and are at least 3 months after the last injection
  • Increased daytime sleepiness (Epworth Sleepiness Scale (ESS\>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.
  • Past or present history of any psychiatric problems, such as schizophrenia, major and/or bipolar depression, or obsessive-compulsive disorder
  • Current use of medication(s) affecting the central nervous system
  • Past or present history of brain and/or laryngeal surgery
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Massachusetts Eye and Ear

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Dysphonia

Interventions

Sodium Oxybate

Condition Hierarchy (Ancestors)

Voice DisordersLaryngeal DiseasesRespiratory Tract DiseasesOtorhinolaryngologic DiseasesNeurologic ManifestationsNervous System DiseasesSigns and SymptomsPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

HydroxybutyratesButyratesAcids, AcyclicCarboxylic AcidsOrganic ChemicalsHydroxy Acids

Central Study Contacts

Kristina Simonyan, MD, PhD, DrMed

CONTACT

6175736025kristina_simonyan@meei.harvard.edu

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Professor of Otolaryngology-Head and Neck Surgery

Study Record Dates

First Submitted

June 4, 2025

First Posted

June 27, 2025

Study Start (Estimated)

October 1, 2026

Primary Completion (Estimated)

September 30, 2027

Study Completion (Estimated)

September 30, 2027

Last Updated

April 28, 2026

Record last verified: 2026-04

Locations

US(1)