Spasmodic DysphoniaPhase 1NCT07041203
Extended-release Sodium Oxybate (Lumryz) in Spasmodic Dysphonia and Voice Tremor
A Phase 1 study testing sodium oxybate in people with Spasmodic Dysphonia and Laryngeal Dystonia, run by Kristina Simonyan at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 21 to 80 years
- Conditions the study is for
- Spasmodic Dysphonia, Laryngeal Dystonia, Voice Tremor
- What is being tested?
- sodium oxybate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Oct 2026to 30 Sep 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
Show all 14Show fewer
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Massachusetts Eye and Ear
Boston, Massachusetts, 02114
Kristina Simonyan, MD, PhD, DrMed, 6175736016, kristina_simonyan@meei.harvard.edu
Kristina Simonyan, MD, PhD, DrMed
About this study
Official title: Central Mechanisms and Treatment Response of Sodium Oxybate (Lumryz) in Spasmodic Dysphonia and Voice Tremor
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.
Read the 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
- Being tested
- sodium oxybate
- Medical subject headings
- Dysphonia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sodium oxybateDrug
Also called Lumryz
Oral administration of sodium oxybate (1.5g, 2.0g, 2.5g, 3.0g)
| Study group | Role | What they receive |
|---|---|---|
| Clinical response to sodium oxybate (Lumryz) | Experimental | Oral administration of sodium oxybate (1.5g, 2.0g, 2.5g, 3.0g) Receives: Drug: sodium oxybate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of Lumryz
The primary outcome will be the percent change from baseline in LD symptoms 60 min after each dose of drug intake
Measured: 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.
Measured: Through study completion, an average of 4 days
Secondary outcomes (1)
Duration of treatment efficacy
The secondary outcomes will be the duration (in hours) of Lumryz treatment efficacy
Measured: Through study completion, an average of 4 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2025
- Study start1 Oct 2026 (estimated)
- Primary completion30 Sep 2027 (estimated)
- Study completion30 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spasmodic DysphoniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kristina Simonyan
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Kristina Simonyan, Massachusetts Eye and Ear Infirmary
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07041203
- Sponsor's protocol ID
- 2025P000919
- Status last verified
- Apr 2026
- First submitted
- 4 Jun 2025
- Last updated
- 28 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Central Mechanisms and Treatment Response of Sodium Oxybate (Lumryz) in Spasmodic Dysphonia and Voice Tremor
- ClinicalTrials.gov ID
- NCT07041203
- Acronym / sponsor ID
- 2025P000919
- Conditions
- Spasmodic Dysphonia, Laryngeal Dystonia, Voice Tremor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Kristina SimonyanUniversity, hospital or other
- Study start
- 1 Oct 2026 (estimated)
- Primary completion
- 30 Sep 2027 (estimated)
- Study completion
- 30 Sep 2027 (estimated)
- First posted
- 27 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Clinical response to sodium oxybate (Lumryz) | Experimental | Drug: sodium oxybateOral administration of sodium oxybate (1.5g, 2.0g, 2.5g, 3.0g) |
Who may qualify
- Ages
- 21 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (14 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| sodium oxybateAlso: LumryzOral administration of sodium oxybate (1.5g, 2.0g, 2.5g, 3.0g) | Drug | Clinical response to sodium oxybate (Lumryz) |
Primary outcome
Efficacy of Lumryz
Time frame: Through study completion, an average of 4 days
The primary outcome will be the percent change from baseline in LD symptoms 60 min after each dose of drug intake
Efficacy of Lumryz
Time frame: Through study completion, an average of 4 days
The primary outcome will be the number of patients (N, % of total) with improved voice symptoms from baseline after each dose of the drug.
1 secondary outcome
Duration of treatment efficacy
Time frame: Through study completion, an average of 4 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing sodium oxybate in people with Spasmodic Dysphonia and Laryngeal Dystonia, run by Kristina Simonyan at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 28 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kristina Simonyan, MD, PhD, DrMed (6175736025, kristina_simonyan@meei.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of Lumryz (time frame: Through study completion, an average of 4 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Apr 2026.