Sodium Oxybate for Spasmodic Dysphonia

Overseen By Kristina Simonyan, MD, PhD, DrMed

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Kristina Simonyan

Must be taking: Sodium oxybate

Must not be taking: CNS medications

Disqualifiers: Pregnancy, Alcoholism, Neurological disorders, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 4 days

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how an extended-release form of sodium oxybate can help with voice symptoms in people with spasmodic dysphonia, a condition causing involuntary spasms in the voice box. Researchers aim to assess the drug's effects on symptoms using behavior tests and brain imaging. Participants with a history of benefiting from immediate-release sodium oxybate and alcohol-responsive laryngeal dystonia (where alcohol temporarily improves voice) might be suitable for this study. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants a chance to be among the first to receive this new form of medication.

Will I have to stop taking my current medications?

The trial requires that you do not use medications affecting the central nervous system while participating.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that extended-release sodium oxybate, known as Lumryz, has been studied for safety in other conditions. In one study, researchers found no significant changes in blood pressure or heart rate, indicating it did not greatly affect these vital signs. Almost all patients in this study were new to oxybate, making these findings particularly relevant for new users.

Another study found that the effects of sodium oxybate lasted up to 300 minutes without any rebound effects, meaning symptoms did not suddenly worsen after the drug wore off. Importantly, there were no reported changes in cognitive function or any signs of self-harm thoughts.

Since sodium oxybate is already approved for treating narcolepsy (a sleep disorder) in a different form, this offers some reassurance about its safety. However, it is important to note that this trial is in an early phase, focusing on safety and determining the right dose for people with spasmodic dysphonia (a voice disorder). Researchers are still collecting data on how well the treatment is tolerated for this specific condition. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for spasmodic dysphonia, which often involve botulinum toxin injections to temporarily paralyze the vocal cords, sodium oxybate offers a potentially new approach by being an oral medication. This extended-release formulation allows for a more controlled release of the drug, possibly leading to more stable symptom management. Researchers are excited about sodium oxybate because it could provide a less invasive and more convenient treatment option, improving the quality of life for patients who currently rely on frequent injections.

What evidence suggests that extended-release sodium oxybate might be an effective treatment for spasmodic dysphonia?

Research has shown that sodium oxybate can help reduce voice problems in people with spasmodic dysphonia. One study found that 82.2% of patients who experienced voice changes after drinking alcohol improved with sodium oxybate. This medication proved much more effective than a placebo in easing symptoms for patients with similar voice conditions. These findings suggest that sodium oxybate, which participants in this trial will receive, may be useful in managing spasmodic dysphonia, especially for those whose symptoms change with alcohol. ^{1 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for individuals with spasmodic dysphonia or voice tremor, a condition that causes involuntary muscle contractions affecting the voice. Participants should be diagnosed with these conditions and willing to undergo clinical and behavioral testing as well as neuroimaging.

Inclusion Criteria

I am between 21 and 80 years old.

Provision of signed and dated informed consent form

I am either male or female.

Exclusion Criteria

I have a learning disability or voice tremor, but no other neurological disorders.

Pregnancy or breastfeeding until a time when they are no longer pregnant or breastfeeding

I am unable to understand and give consent for treatment.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral administration of sodium oxybate (Lumryz) in varying doses to assess efficacy and safety

4 days

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Extended-release Sodium Oxybate

Trial Overview The study is testing an extended-release form of sodium oxybate on how it affects voice symptoms in people with spasmodic dysphonia. It's an open-label study, meaning everyone knows what treatment they're getting, and it aims to find the right dose.

How Is the Trial Designed?

Group I: Clinical response to sodium oxybate (Lumryz) Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Kristina Simonyan

Lead Sponsor

Trials

4

Recruited

740+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07041203

Extended-release Sodium Oxybate (Lumryz) in Spasmodic ... 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 ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT07041203?term=AREA%5BBasicSearch%5D(oxybate)&rank=5

Extended-release Sodium Oxybate (Lumryz) in Spasmodic ... A type of clinical study in which participants are identified as belonging to study groups and are assessed for biomedical or health outcomes. Participants may ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5415435/

An open-label study of sodium oxybate (Xyrem®) in ... Our study demonstrated that sodium oxybate reduced voice symptoms in 82.2% of alcohol-responsive SD patients both with and without co-occurring VT.

4. clinicaladvisor.com clinicaladvisor.com/news/sodium-oxybate-treatment-focal-laryngeal-dystonia/

Sodium Oxybate Shows Promise in Treatment of Focal ... Sodium oxybate was significantly more effective at reducing symptoms than placebo for patients with alcohol-responsive LD.

5. app.trialscreen.org app.trialscreen.org/trials/phase-1-extended-release-sodium-oxybate-lumryz-spasmodic-dysphonia-voice-trial-nct07041203

Extended-release Sodium Oxybate (Lumryz) in Spasmodic ... Benefits: Participation in this study may provide relief from voice symptoms for people with alcohol-responsive SD, as previous studies have shown that ...

6. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/full/10.1002/ana.27121

Efficacy and Safety of Sodium Oxybate in Isolated Focal ... Drug efficacy waned by 300 minutes after intake without a rebound. No changes were found in cognitive function, suicidality, or vital signs.

7. lumryzhcp.com lumryzhcp.com/clinical-results/lumryz-safety

Safety | LUMRYZ™ (sodium oxybate) for extended-release ... There were no clinically meaningful changes from baseline blood pressure or heart rate. *All but 1 patient included in the safety study were oxybate-naïve. In ...

8. rally.massgeneralbrigham.org rally.massgeneralbrigham.org/study/xyrem

Sodium oxybate as a novel oral drug for spasmodic ... The purpose of this research study to learn how a new oral drug, sodium oxybate (Xyrem), improves symptoms of spasmodic dysphonia.

9. go.drugbank.com go.drugbank.com/drugs/DB09072

Sodium oxybate: Uses, Interactions, Mechanism of Action Sodium oxybate (Xyrem) is a central nervous system (CNS) depressant used to treat cataplexy or excessive daytime sleepiness associated with narcolepsy.