Evidence map›Paper›PMID 39565101›Full record

Trial reportAnnals of neurology2025

Efficacy and Safety of Sodium Oxybate in Isolated Focal Laryngeal Dystonia: A Phase IIb Double-Blind Placebo-Controlled Cross-Over Randomized Clinical Trial.

Kristina Simonyan, Lena C O'Flynn, Azadeh Hamzehei Sichani, Steven J Frucht, Anna F Rumbach, Nutan Sharma, Phillip C Song, Alexis Worthley

Abstract readClinical Trial, Phase IIRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Annals of neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

6 citing papers in PubMed.

  1. Brain-Computer Interface Improves Symptoms of Isolated Focal Laryngeal Dystonia: A Single-Blind Study.Movement disorders : official journal of the Movement Disorder Society · 2026
    Article
  2. Article
  3. Treatment of Neurogenic Voice Disorders.World journal of otorhinolaryngology - head and neck surgery · 2025
    Review
  4. Spasmodic Dysphonia.World journal of otorhinolaryngology - head and neck surgery · 2025
    Review
  5. Article
  6. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Kristina SimonyanDepartment of Otolaryngology-Head and Neck Surgery, Massachusetts Eye and Ear and Harvard Medical School, Boston, MA.ORCID 0000-0001-7444-0437
Lena C O'FlynnDepartment of Otolaryngology-Head and Neck Surgery, Massachusetts Eye and Ear and Harvard Medical School, Boston, MA.
Azadeh Hamzehei SichaniDepartment of Otolaryngology-Head and Neck Surgery, Massachusetts Eye and Ear and Harvard Medical School, Boston, MA.
Steven J FruchtDepartment of Neurology, NYU Langone Health, New York, NY.
Anna F RumbachSpeech Pathology, School of Health and Rehabilitation Sciences, The University of Queensland, Brisbane, Australia.
Nutan SharmaDepartment of Neurology, Massachusetts General Hospital, Boston, MA.
Phillip C SongDepartment of Otolaryngology-Head and Neck Surgery, Massachusetts Eye and Ear and Harvard Medical School, Boston, MA.
Alexis WorthleyDepartment of Otolaryngology-Head and Neck Surgery, Massachusetts Eye and Ear and Harvard Medical School, Boston, MA.

Funding

Voice tremor in spasmodic dysphonia: central mechanisms and treatment responseR01DC012545 · NIDCD · MASSACHUSETTS EYE AND EAR INFIRMARY · PI SIMONYAN, KRISTINA · 2012 to 2021
$5.2M
NIDCD NIH HHS R01 DC012545NIDCD NIH HHS R01DC012545
6 · The paper itself

Abstract

objectiveTo examine the efficacy and safety of sodium oxybate versus placebo in a phase IIb randomized double-blind placebo-controlled 2-period cross-over clinical trial in patients with isolated laryngeal dystonia (LD).

methodsThe study was conducted from January 2018 to December 2021, pausing during the COVID-19 pandemic, at Massachusetts Eye and Ear in 106 patients with alcohol-responsive (EtOH+) and alcohol-non-responsive (EtOH-) LD (53 to receive 1.5g of sodium oxybate first, 53 to receive matching placebo first). The primary outcome was a change from baseline in LD symptom severity 40 minutes after drug intake. Safety was based on vital signs, cognitive function, suicidality, daytime sleepiness, and adverse events. Patients, investigators, and outcome assessors were masked to study procedures.

resultsCompared to baseline, EtOH+ but not EtOH- patients had a statistically significant improvement in LD symptoms following sodium oxybate versus placebo (EtOH+: 98.75% confidence interval [CI] = 0.6-26.9; p = 0.008; EtOH-: 98.75% CI = -6.2 to 18.7; p = 0.42). Statistically significant minimum drug efficacy in EtOH+ patients was found at ≥16% symptom improvement (OR = 2.09; 98.75% CI = 0.75-5.80; p = 0.036), with an average of 40.81% benefits (98.75% CI = 34.7-48.6). Drug efficacy waned by 300 minutes after intake without a rebound. No changes were found in cognitive function, suicidality, or vital signs. Common adverse events included mild dizziness, nausea, and daytime sleepiness.

interpretationSodium oxybate showed clinically meaningful improvement of symptoms in EtOH+ LD patients, with acceptable tolerability. Sodium oxybate offers the first pathophysiologically relevant oral treatment for laryngeal dystonia. ANN NEUROL 2025;97:329-343.

Indexed as

Dystonic DisordersLaryngeal DiseasesSodium OxybateAdultAgedCross-Over StudiesDouble-Blind MethodFemaleHumansMaleMiddle AgedTreatment OutcomeSodium Oxybate

Identifiers

PMID39565101
PMCPMC11740276

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.