Viewing Study NCT03292458

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Ignite Creation Date: 2025-12-25 @ 12:00 AM
Ignite Modification Date: 2026-03-03 @ 2:21 AM
Study NCT ID: NCT03292458
Status: COMPLETED
Last Update Posted: 2025-04-04
First Post: 2017-09-20
Is Gene Therapy: True
Has Adverse Events: True
Brief Title: Sodium Oxybate in Spasmodic Dysphonia and Voice Tremor
Sponsor:
Organization:
Overview Dates Organization Conditions Design Enrollment Locations Outcomes Modules Raw JSON

Raw JSON

{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2024-12-13', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'D055154', 'term': 'Dysphonia'}], 'ancestors': [{'id': 'D014832', 'term': 'Voice Disorders'}, {'id': 'D007818', 'term': 'Laryngeal Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D010038', 'term': 'Otorhinolaryngologic Diseases'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D012978', 'term': 'Sodium Oxybate'}, {'id': 'D000431', 'term': 'Ethanol'}], 'ancestors': [{'id': 'D006885', 'term': 'Hydroxybutyrates'}, {'id': 'D002087', 'term': 'Butyrates'}, {'id': 'D000144', 'term': 'Acids, Acyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D006880', 'term': 'Hydroxy Acids'}, {'id': 'D000438', 'term': 'Alcohols'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'kristina_simonyan@meei.harvard.edu', 'phone': '617-573-6016', 'title': 'Prof. Kristina Simonyan', 'organization': 'Massachusetts Eye and Ear'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}, 'limitationsAndCaveats': {'description': 'A single-center trial: However, the patients were recruited across the US, the UK, and Australia, thus expanding the generalizability of the findings.\n\nAn inclusion of a greater number of Non-Hispanic White females: LD is well-documented to affect the Non-Hispanic White population with a 4:1 female to male prevalence. Thus, the demographics of patients recruited for this trial reflect the clinical demographics of the disorder in general.'}}, 'adverseEventsModule': {'timeFrame': '5 hours and a 24-hour follow-up', 'description': 'Safety was documented as a change from baseline in vital signs (blood pressure, pulse rate), cognitive function (MoCA), suicidality (C-SSRS), and daytime sleepiness (Epworth sleepiness scale). Adverse events were assessed using a structured side effects questionnaire and unstructured patient reporting (unprompted self-reporting by patients) as none, mild, moderate, or severe.\n\nPre-specified to report adverse events by alcohol responsiveness and intervention only.', 'eventGroups': [{'id': 'EG000', 'title': 'Sodium Oxybate: Alcohol-responsive (EtOH+) Laryngeal Dystonia', 'description': 'For analysis, patients in groups (1) Alcohol-responsive (EtOH+) Laryngeal Dystonia and (2) Alcohol-responsive (EtOH+) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH+ group.', 'otherNumAtRisk': 50, 'deathsNumAtRisk': 50, 'otherNumAffected': 29, 'seriousNumAtRisk': 50, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Placebo: Alcohol-responsive (EtOH+) Laryngeal Dystonia', 'description': 'For analysis, patients in groups (1) Alcohol-responsive (EtOH+) Laryngeal Dystonia and (2) Alcohol-responsive (EtOH+) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH+ group.', 'otherNumAtRisk': 50, 'deathsNumAtRisk': 50, 'otherNumAffected': 8, 'seriousNumAtRisk': 50, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'Sodium Oxybate: Alcohol-non-responsive (EtOH-) Laryngeal Dystonia', 'description': 'For analysis, patients in groups (3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia and (4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH- group.', 'otherNumAtRisk': 56, 'deathsNumAtRisk': 56, 'otherNumAffected': 37, 'seriousNumAtRisk': 56, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG003', 'title': 'Placebo: Alcohol-non-responsive (EtOH-) Laryngeal Dystonia', 'description': 'For analysis, patients in groups (3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia and (4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH- group.', 'otherNumAtRisk': 56, 'deathsNumAtRisk': 56, 'otherNumAffected': 13, 'seriousNumAtRisk': 56, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Mild Dizziness/ lightheadedness/unsteadiness/clumsiness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 24}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 27}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 8}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Blurred vision', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 4}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Trouble concentrating', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Confusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Restlessness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Disorientation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Feeling hangover', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Nervousness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Lethargy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 8}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Dry mouth', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Stomach pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Mild Daytime sleepiness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 8}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Moderate Dizziness/ lightheadedness/unsteadiness/clumsiness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Moderate Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 50, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 50, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 56, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 56, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Symptom Severity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '50', 'groupId': 'OG000'}, {'value': '56', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Alcohol-responsive (EtOH+) Laryngeal Dystonia', 'description': 'Laryngeal dystonia with and without dystonic tremor of voice'}, {'id': 'OG001', 'title': 'Alcohol-non-responsive (EtOH-) Laryngeal Dystonia', 'description': 'Laryngeal dystonia with and without dystonic tremor of voice'}], 'classes': [{'title': 'Sodium oxybate', 'categories': [{'measurements': [{'value': '28.0', 'groupId': 'OG000', 'lowerLimit': '21.0', 'upperLimit': '35.0'}, {'value': '18.6', 'groupId': 'OG001', 'lowerLimit': '12.0', 'upperLimit': '25.2'}]}]}, {'title': 'Placebo', 'categories': [{'measurements': [{'value': '14.2', 'groupId': 'OG000', 'lowerLimit': '7.2', 'upperLimit': '21.2'}, {'value': '12.3', 'groupId': 'OG001', 'lowerLimit': '5.7', 'upperLimit': '19.0'}]}]}], 'analyses': [{'pValue': '0.01', 'groupIds': ['OG000', 'OG001'], 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '98.75', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY', 'nonInferiorityComment': 'The primary efficacy outcome was analyzed using a restricted maximum likelihood (REML)-based linear mixed-effect model. The analysis included group, treatment, and treatment period as interacting fixed effects and subject as random effect. An unstructured covariance structure was used to model the within-patient errors.'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': '40 min after drug or placebo administration', 'description': 'A combined clinician-objective and patient-subjective change in visual analog scale (min-max 0-100, a higher score is the worse outcome) score of symptom severity before and after drug vs. placebo intake.\n\nFor analysis, patients in groups (1) Alcohol-responsive (EtOH+) Laryngeal Dystonia and (2) Alcohol-responsive (EtOH+) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH+ group.\n\nFor analysis, patients in groups (3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia and (4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH- group.', 'unitOfMeasure': 'score on a scale', 'dispersionType': '98.75% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Minimum Treatment Efficacy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '50', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Alcohol-responsive (EtOH+) Laryngeal Dystonia', 'description': 'Laryngeal dystonia with and without dystonic tremor of voice. Pre-specified to analysis based only on alcohol responsiveness (EtOH+).'}], 'classes': [{'title': 'Symptom improvement ≥ 16%', 'denoms': [{'units': 'Participants', 'counts': [{'value': '32', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '40.81', 'groupId': 'OG000', 'lowerLimit': '34.7', 'upperLimit': '48.6'}]}]}, {'title': 'Symptom improvement < 16%', 'denoms': [{'units': 'Participants', 'counts': [{'value': '18', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.76', 'groupId': 'OG000', 'lowerLimit': '1.1', 'upperLimit': '8.1'}]}]}], 'analyses': [{'pValue': '0.05', 'groupIds': ['OG000'], 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '98.75', 'groupDescription': 'The minimum and average efficacy of sodium oxybate vs. placebo in improving symptoms compared to the baseline were determined using binomial logistic regression.', 'statisticalMethod': 'Regression, Logistic', 'nonInferiorityType': 'OTHER', 'nonInferiorityComment': 'The minimum and average efficacy (in % symptom change) of sodium oxybate versus placebo in improving symptoms compared to the baseline was determined using binomial logistic regression.'}], 'paramType': 'MEAN', 'timeFrame': '40 min after drug or placebo administration', 'description': 'Pre-specified to analysis based only on alcohol responsiveness (EtOH+). A combined clinician-objective and patient-subjective change in visual analog scale score (min-max 0-100, a higher score is the worse outcome) of symptom severity in EtOH+ patients.', 'unitOfMeasure': 'percentage of symptom change', 'dispersionType': '98.75% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Pre-specified to only report Alcohol-responsive (EtOH+) Laryngeal Dystonia Arm'}, {'type': 'SECONDARY', 'title': 'Treatment Efficacy Dependent on LD Clinical Type', 'denoms': [{'units': 'Participants', 'counts': [{'value': '23', 'groupId': 'OG000'}, {'value': '27', 'groupId': 'OG001'}, {'value': '30', 'groupId': 'OG002'}, {'value': '26', 'groupId': 'OG003'}, {'value': '25', 'groupId': 'OG004'}, {'value': '20', 'groupId': 'OG005'}, {'value': '33', 'groupId': 'OG006'}, {'value': '21', 'groupId': 'OG007'}]}], 'groups': [{'id': 'OG000', 'title': 'Alcohol-responsive (EtOH+) Laryngeal Dystonia', 'description': 'Laryngeal dystonia'}, {'id': 'OG001', 'title': 'Alcohol-responsive (EtOH+) Laryngeal Dystonia With Dystonic Tremor of Voice', 'description': 'Laryngeal dystonia with dystonic tremor of voice'}, {'id': 'OG002', 'title': 'Alcohol-non-responsive (EtOH-) Laryngeal Dystonia', 'description': 'Laryngeal dystonia'}, {'id': 'OG003', 'title': 'Alcohol-non-responsive (EtOH-) Laryngeal Dystonia With Dystonic Tremor of Voice', 'description': 'Laryngeal dystonia with dystonic tremor of voice'}, {'id': 'OG004', 'title': 'Alcohol-responsive Adductor Type of Laryngeal Dystonia', 'description': 'Laryngeal dystonia, adductor type'}, {'id': 'OG005', 'title': 'Alcohol-responsive Abductor Type of Laryngeal Dystonia', 'description': 'Laryngeal dystonia, abductor type'}, {'id': 'OG006', 'title': 'Alcohol-non-responsive Adductor Type of Laryngeal Dystonia', 'description': 'Laryngeal dystonia, adductor type'}, {'id': 'OG007', 'title': 'Alcohol-non-responsive Abductor Type of Laryngeal Dystonia', 'description': 'Laryngeal dystonia, abductor type'}], 'classes': [{'title': 'Sodium oxybate', 'categories': [{'measurements': [{'value': '24.65', 'spread': '4.45', 'groupId': 'OG000'}, {'value': '30.54', 'spread': '4.29', 'groupId': 'OG001'}, {'value': '18.30', 'spread': '3.07', 'groupId': 'OG002'}, {'value': '18.96', 'spread': '3.49', 'groupId': 'OG003'}, {'value': '31.70', 'spread': '5.08', 'groupId': 'OG004'}, {'value': '24.85', 'spread': '3.92', 'groupId': 'OG005'}, {'value': '21.36', 'spread': '2.86', 'groupId': 'OG006'}, {'value': '14.96', 'spread': '4.04', 'groupId': 'OG007'}]}]}, {'title': 'Placebo', 'categories': [{'measurements': [{'value': '18.07', 'spread': '3.99', 'groupId': 'OG000'}, {'value': '10.85', 'spread': '5.90', 'groupId': 'OG001'}, {'value': '10.34', 'spread': '1.94', 'groupId': 'OG002'}, {'value': '14.47', 'spread': '1.93', 'groupId': 'OG003'}, {'value': '13.59', 'spread': '6.07', 'groupId': 'OG004'}, {'value': '13.10', 'spread': '4.62', 'groupId': 'OG005'}, {'value': '13.07', 'spread': '1.54', 'groupId': 'OG006'}, {'value': '11.32', 'spread': '2.83', 'groupId': 'OG007'}]}]}], 'analyses': [{'pValue': '0.01', 'groupIds': ['OG000', 'OG001', 'OG002', 'OG003', 'OG004', 'OG005', 'OG006', 'OG007'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'paramValue': '98.75', 'statisticalMethod': 't-test, 2 sided', 'nonInferiorityType': 'OTHER'}], 'paramType': 'MEAN', 'timeFrame': '40 min after drug or placebo administration', 'description': 'A combined clinician-objective and patient-subjective change in visual analog scale (min-max 0-100, a higher score is the worse outcome) score of symptom severity before and after drug vs. placebo intake.\n\nEtOH+ and EtOH- were stratified based on phenotypical characteristics, including LD, LD with Dystonic Tremor of Voice, Abductor LD, Adductor LD.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Length of Treatment Efficacy', 'denoms': [{'units': 'Participants', 'counts': [{'value': '50', 'groupId': 'OG000'}, {'value': '56', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Alcohol-responsive (EtOH+) Laryngeal Dystonia', 'description': 'Laryngeal dystonia with and without dystonic tremor of voice'}, {'id': 'OG001', 'title': 'Alcohol-non-responsive (EtOH-) Laryngeal Dystonia', 'description': 'Laryngeal dystonia with and without dystonic tremor of voice'}], 'classes': [{'title': 'Sodium oxybate: 40 min', 'categories': [{'measurements': [{'value': '28.0', 'spread': '3.06', 'groupId': 'OG000'}, {'value': '18.60', 'spread': '2.31', 'groupId': 'OG001'}]}]}, {'title': 'Sodium oxybate: 180 min', 'categories': [{'measurements': [{'value': '18.63', 'spread': '3.36', 'groupId': 'OG000'}, {'value': '12.67', 'spread': '2.40', 'groupId': 'OG001'}]}]}, {'title': 'Sodium oxybate: 300 min', 'categories': [{'measurements': [{'value': '15.47', 'spread': '4.32', 'groupId': 'OG000'}, {'value': '8.33', 'spread': '1.98', 'groupId': 'OG001'}]}]}, {'title': 'Placebo: 40 min', 'categories': [{'measurements': [{'value': '14.23', 'spread': '3.71', 'groupId': 'OG000'}, {'value': '12.34', 'spread': '1.39', 'groupId': 'OG001'}]}]}, {'title': 'Placebo: 180 min', 'categories': [{'measurements': [{'value': '9.26', 'spread': '3.61', 'groupId': 'OG000'}, {'value': '9.31', 'spread': '1.99', 'groupId': 'OG001'}]}]}, {'title': 'Placebo: 300 min', 'categories': [{'measurements': [{'value': '10.30', 'spread': '3.15', 'groupId': 'OG000'}, {'value': '8.16', 'spread': '1.84', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.01', 'groupIds': ['OG000', 'OG001'], 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '98.75', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'OTHER'}], 'paramType': 'MEAN', 'timeFrame': '40 min to 5 hours after drug or placebo administration', 'description': 'The length of treatment efficacy in each EtOH+ and EtOH- group was assessed at 40, 180, and 300 min after drug vs. placebo intake compared to the baseline using a combined clinician-objective and patient-subjective change in visual analog scale (min-max 0-100, a higher score is the worse outcome) score of symptom severity.\n\nFor analysis, patients in groups (1) Alcohol-responsive (EtOH+) Laryngeal Dystonia and (2) Alcohol-responsive (EtOH+) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH+ group.\n\nFor analysis, patients in groups (3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia and (4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH- group.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Pre-specified to only report based on alcohol-responsiveness (EtOH+, EtOH-)'}, {'type': 'SECONDARY', 'title': 'Relationship Between Alcohol-responsiveness of Symptoms and Drug-induced Symptom Improvement', 'denoms': [{'units': 'Participants', 'counts': [{'value': '50', 'groupId': 'OG000'}, {'value': '56', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Alcohol-responsive (EtOH+) Laryngeal Dystonia', 'description': 'Laryngeal dystonia with and without dystonic tremor of voice'}, {'id': 'OG001', 'title': 'Alcohol-non-responsive (EtOH-) Laryngeal Dystonia', 'description': 'Laryngeal dystonia with and without dystonic tremor of voice'}], 'classes': [{'title': 'Sodium oxybate', 'categories': [{'measurements': [{'value': '0.45', 'groupId': 'OG000', 'lowerLimit': '0.10', 'upperLimit': '6.88'}, {'value': '0.09', 'groupId': 'OG001', 'lowerLimit': '-0.024', 'upperLimit': '0.35'}]}]}, {'title': 'Placebo', 'categories': [{'measurements': [{'value': '0.06', 'groupId': 'OG000', 'lowerLimit': '-0.034', 'upperLimit': '0.39'}, {'value': '0.22', 'groupId': 'OG001', 'lowerLimit': '-0.10', 'upperLimit': '0.55'}]}]}], 'analyses': [{'pValue': '0.01', 'groupIds': ['OG000', 'OG001'], 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '98.75', 'statisticalMethod': 'Pearson correlation', 'nonInferiorityType': 'OTHER'}], 'paramType': 'NUMBER', 'timeFrame': '40 min after drug or placebo administration', 'description': 'A combined clinician-objective and patient-subjective change in visual analog scale (min-max 0-100, a higher score is the worse outcome) score of symptom severity before and 40 min after drug vs. placebo intake.\n\nFor analysis, patients in groups (1) Alcohol-responsive (EtOH+) Laryngeal Dystonia and (2) Alcohol-responsive (EtOH+) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH+ group.\n\nFor analysis, patients in groups (3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia and (4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH- group.', 'unitOfMeasure': 'correlation coefficient', 'dispersionType': '98.75% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Pre-specified to only report based on alcohol-responsiveness (EtOH+, EtOH-)'}, {'type': 'SECONDARY', 'title': 'Relationship Between Symptom Severity Change and Dystonia Clinical Characteristics', 'denoms': [{'units': 'Participants', 'counts': [{'value': '50', 'groupId': 'OG000'}, {'value': '56', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Alcohol-responsive (EtOH+) Laryngeal Dystonia', 'description': 'Laryngeal dystonia with and without dystonic tremor of voice'}, {'id': 'OG001', 'title': 'Alcohol-non-responsive (EtOH-) Laryngeal Dystonia', 'description': 'Laryngeal dystonia with and without dystonic tremor of voice'}], 'classes': [{'title': 'Sodium oxybate: duration', 'categories': [{'measurements': [{'value': '-0.17', 'groupId': 'OG000', 'lowerLimit': '-0.51', 'upperLimit': '0.16'}, {'value': '0.10', 'groupId': 'OG001', 'lowerLimit': '-0.26', 'upperLimit': '0.49'}]}]}, {'title': 'Placebo: duration', 'categories': [{'measurements': [{'value': '0.18', 'groupId': 'OG000', 'lowerLimit': '-0.21', 'upperLimit': '0.52'}, {'value': '0.16', 'groupId': 'OG001', 'lowerLimit': '-0.17', 'upperLimit': '0.44'}]}]}, {'title': 'Sodium oxybate: age of symptom onset', 'categories': [{'measurements': [{'value': '0.16', 'groupId': 'OG000', 'lowerLimit': '-0.14', 'upperLimit': '0.47'}, {'value': '-0.15', 'groupId': 'OG001', 'lowerLimit': '-0.43', 'upperLimit': '0.18'}]}]}, {'title': 'Placebo: age of symptom onset', 'categories': [{'measurements': [{'value': '-0.18', 'groupId': 'OG000', 'lowerLimit': '-0.46', 'upperLimit': '0.22'}, {'value': '-0.09', 'groupId': 'OG001', 'lowerLimit': '-0.40', 'upperLimit': '0.30'}]}]}, {'title': 'Sodium oxybate: baseline symptom severity', 'categories': [{'measurements': [{'value': '-0.05', 'groupId': 'OG000', 'lowerLimit': '-0.31', 'upperLimit': '0.20'}, {'value': '-0.04', 'groupId': 'OG001', 'lowerLimit': '-0.47', 'upperLimit': '0.36'}]}]}, {'title': 'Placebo: baseline symptom severity', 'categories': [{'measurements': [{'value': '0.24', 'groupId': 'OG000', 'lowerLimit': '-0.20', 'upperLimit': '0.58'}, {'value': '-0.11', 'groupId': 'OG001', 'lowerLimit': '-0.39', 'upperLimit': '0.18'}]}]}], 'analyses': [{'pValue': '0.01', 'groupIds': ['OG000', 'OG001'], 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '98.75', 'statisticalMethod': 'Pearson correlation', 'nonInferiorityType': 'OTHER'}], 'paramType': 'NUMBER', 'timeFrame': '40 min after drug or placebo administration', 'description': 'A combined clinician-objective and patient-subjective change in visual analog scale (min-max 0-100, a higher score is the worse outcome) score of symptom severity before and 40 min after drug vs. placebo intake.\n\nFor analysis, patients in groups (1) Alcohol-responsive (EtOH+) Laryngeal Dystonia and (2) Alcohol-responsive (EtOH+) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH+ group.\n\nFor analysis, patients in groups (3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia and (4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH- group.', 'unitOfMeasure': 'correlation coefficient', 'dispersionType': '98.75% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Pre-specified to only report based on alcohol-responsiveness (EtOH+, EtOH-)'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Laryngeal Dystonia With and Without Dystonic Tremor of Voice: Sodium Oxybate, Then Placebo', 'description': 'Participants with alcohol-responsive and non-responsive LD with/without dystonic tremor of voice first received a single dose of oral solution of sodium oxybate 1.5 g. After a 24-hour washout period, they then received oral solution of placebo (matching sodium oxybate 1.5 g).\n\nAccording to a priori hypothesis, the analysis was conducted based on the alcohol-responsiveness of symptoms, regardless of period sequence.\n\nBaseline measures are described based on LD phenotype and alcohol-responsiveness for detailed cohort description.'}, {'id': 'FG001', 'title': 'Laryngeal Dystonia With and Without Dystonic Tremor of Voice: Placebo, Then Sodium Oxybate', 'description': 'Participants with alcohol-responsive and non-responsive LD with/without dystonic tremor of voice first received a single dose of oral solution of placebo (matching sodium oxybate 1.5 g). After a 24-hour washout period, they then received oral solution of sodium oxybate 1.5 g.\n\nAccording to a priori hypothesis, the analysis was conducted based on the alcohol-responsiveness of symptoms, regardless of period sequence.\n\nBaseline measures are described based on LD phenotype and alcohol-responsiveness for detailed cohort description.'}], 'periods': [{'title': 'Period 1', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '55'}, {'groupId': 'FG001', 'numSubjects': '53'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '53'}, {'groupId': 'FG001', 'numSubjects': '53'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}, {'title': 'Period 2', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '53'}, {'groupId': 'FG001', 'numSubjects': '53'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '53'}, {'groupId': 'FG001', 'numSubjects': '53'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'The study was conducted between January 22, 2018, and December 29, 2021. The COVID-19 pandemic and the associated lockdown of human research activities fully paused patient recruitment and the conduct of study procedures between March 2020 and December 2020.', 'preAssignmentDetails': 'Pre-specified to LD patients with and without dystonic voice tremor regardless of alcohol responsiveness of symptoms.\n\n9 patients were excluded because of a history of suicidal ideations (n= 3), absence of symptoms at the time of study participation (n= 3), left-handedness (n= 1), presence of multifocal dystonia (n= 1), and contraindications to MRI (n= 1).'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '23', 'groupId': 'BG000'}, {'value': '29', 'groupId': 'BG001'}, {'value': '30', 'groupId': 'BG002'}, {'value': '26', 'groupId': 'BG003'}, {'value': '108', 'groupId': 'BG004'}]}], 'groups': [{'id': 'BG000', 'title': '(1) Alcohol-responsive (EtOH+) Laryngeal Dystonia', 'description': 'For analysis, patients in groups (1) Alcohol-responsive (EtOH+) Laryngeal Dystonia and (2) Alcohol-responsive (EtOH+) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH+ group.'}, {'id': 'BG001', 'title': '(2) Alcohol-responsive (EtOH+) Laryngeal Dystonia With Dystonic Tremor of Voice', 'description': 'For analysis, patients in groups (1) Alcohol-responsive (EtOH+) Laryngeal Dystonia and (2) Alcohol-responsive (EtOH+) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH+ group.'}, {'id': 'BG002', 'title': '(3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia', 'description': 'For analysis, patients in groups (3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia and (4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH- group.'}, {'id': 'BG003', 'title': '(4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia With Dystonic Tremor of Voice', 'description': 'For analysis, patients in groups (3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia and (4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH- group.'}, {'id': 'BG004', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '19', 'groupId': 'BG000'}, {'value': '16', 'groupId': 'BG001'}, {'value': '23', 'groupId': 'BG002'}, {'value': '12', 'groupId': 'BG003'}, {'value': '70', 'groupId': 'BG004'}]}, {'title': '>=65 years', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}, {'value': '14', 'groupId': 'BG003'}, {'value': '38', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '56.0', 'groupId': 'BG000', 'lowerLimit': '48.5', 'upperLimit': '61.5'}, {'value': '62.0', 'groupId': 'BG001', 'lowerLimit': '52.0', 'upperLimit': '68.0'}, {'value': '57.0', 'groupId': 'BG002', 'lowerLimit': '48.0', 'upperLimit': '63.8'}, {'value': '66.5', 'groupId': 'BG003', 'lowerLimit': '54.3', 'upperLimit': '71.8'}, {'value': '59.5', 'groupId': 'BG004', 'lowerLimit': '50.0', 'upperLimit': '68.0'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'INTER_QUARTILE_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '15', 'groupId': 'BG000'}, {'value': '26', 'groupId': 'BG001'}, {'value': '17', 'groupId': 'BG002'}, {'value': '18', 'groupId': 'BG003'}, {'value': '76', 'groupId': 'BG004'}]}, {'title': 'Male', 'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '13', 'groupId': 'BG002'}, {'value': '8', 'groupId': 'BG003'}, {'value': '32', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '23', 'groupId': 'BG000'}, {'value': '29', 'groupId': 'BG001'}, {'value': '30', 'groupId': 'BG002'}, {'value': '26', 'groupId': 'BG003'}, {'value': '108', 'groupId': 'BG004'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Black or African American', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}, {'value': '7', 'groupId': 'BG004'}]}, {'title': 'White', 'measurements': [{'value': '20', 'groupId': 'BG000'}, {'value': '28', 'groupId': 'BG001'}, {'value': '29', 'groupId': 'BG002'}, {'value': '24', 'groupId': 'BG003'}, {'value': '101', 'groupId': 'BG004'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Handedness', 'classes': [{'categories': [{'title': 'Right-handed', 'measurements': [{'value': '23', 'groupId': 'BG000'}, {'value': '29', 'groupId': 'BG001'}, {'value': '30', 'groupId': 'BG002'}, {'value': '26', 'groupId': 'BG003'}, {'value': '108', 'groupId': 'BG004'}]}, {'title': 'Left-handed', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Monolingual native English Language', 'classes': [{'categories': [{'measurements': [{'value': '23', 'groupId': 'BG000'}, {'value': '29', 'groupId': 'BG001'}, {'value': '30', 'groupId': 'BG002'}, {'value': '26', 'groupId': 'BG003'}, {'value': '108', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Cognitive function', 'classes': [{'categories': [{'title': '≥ 26 points (normal cognitive function)', 'measurements': [{'value': '23', 'groupId': 'BG000'}, {'value': '29', 'groupId': 'BG001'}, {'value': '30', 'groupId': 'BG002'}, {'value': '26', 'groupId': 'BG003'}, {'value': '108', 'groupId': 'BG004'}]}, {'title': '< 26 points (impaired cognitive function)', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Montreal Cognitive Assessment Test Score', 'unitOfMeasure': 'Participants'}, {'title': 'Dystonia phenotype', 'classes': [{'categories': [{'title': 'Adductor type', 'measurements': [{'value': '12', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '17', 'groupId': 'BG002'}, {'value': '16', 'groupId': 'BG003'}, {'value': '58', 'groupId': 'BG004'}]}, {'title': 'Abductor type', 'measurements': [{'value': '10', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}, {'value': '43', 'groupId': 'BG004'}]}, {'title': 'Mixed adductor and abductor type', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}, {'value': '7', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Family history of dystonia', 'classes': [{'categories': [{'title': 'Familial type', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}, {'value': '8', 'groupId': 'BG004'}]}, {'title': 'Sporadic type', 'measurements': [{'value': '20', 'groupId': 'BG000'}, {'value': '27', 'groupId': 'BG001'}, {'value': '28', 'groupId': 'BG002'}, {'value': '25', 'groupId': 'BG003'}, {'value': '100', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age of onset', 'classes': [{'categories': [{'measurements': [{'value': '38.0', 'groupId': 'BG000', 'lowerLimit': '31.5', 'upperLimit': '46.0'}, {'value': '50.0', 'groupId': 'BG001', 'lowerLimit': '32.0', 'upperLimit': '56.0'}, {'value': '43.5', 'groupId': 'BG002', 'lowerLimit': '32.5', 'upperLimit': '52.8'}, {'value': '46.5', 'groupId': 'BG003', 'lowerLimit': '31.0', 'upperLimit': '60.0'}, {'value': '44.0', 'groupId': 'BG004', 'lowerLimit': '31.8', 'upperLimit': '54.0'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'INTER_QUARTILE_RANGE'}, {'title': 'Dystonia duration', 'classes': [{'categories': [{'measurements': [{'value': '13.0', 'groupId': 'BG000', 'lowerLimit': '9.5', 'upperLimit': '20.0'}, {'value': '13.0', 'groupId': 'BG001', 'lowerLimit': '8.0', 'upperLimit': '22.0'}, {'value': '11.0', 'groupId': 'BG002', 'lowerLimit': '5.3', 'upperLimit': '23.5'}, {'value': '13.4', 'groupId': 'BG003', 'lowerLimit': '7.3', 'upperLimit': '28.0'}, {'value': '13.0', 'groupId': 'BG004', 'lowerLimit': '7.0', 'upperLimit': '22.0'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'INTER_QUARTILE_RANGE'}, {'title': 'Botulinum toxin treatment', 'classes': [{'categories': [{'measurements': [{'value': '20', 'groupId': 'BG000'}, {'value': '26', 'groupId': 'BG001'}, {'value': '26', 'groupId': 'BG002'}, {'value': '21', 'groupId': 'BG003'}, {'value': '93', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Centrally acting medications', 'classes': [{'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}, {'value': '5', 'groupId': 'BG003'}, {'value': '24', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'Pre-specified to analysis based only on the diagnosis (LD, LD/DTv) and alcohol responsiveness (EtOH+, EtOH-).'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2024-03-04', 'size': 709042, 'label': 'Study Protocol, Statistical Analysis Plan, and Informed Consent Form', 'hasIcf': True, 'hasSap': True, 'filename': 'Prot_SAP_ICF_002.pdf', 'typeAbbrev': 'Prot_SAP_ICF', 'uploadDate': '2025-02-06T21:23', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'BASIC_SCIENCE', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 117}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-01-22', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-04', 'completionDateStruct': {'date': '2024-10-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-04-03', 'studyFirstSubmitDate': '2017-09-20', 'resultsFirstSubmitDate': '2024-11-19', 'studyFirstSubmitQcDate': '2017-09-20', 'lastUpdatePostDateStruct': {'date': '2025-04-04', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2025-04-03', 'studyFirstPostDateStruct': {'date': '2017-09-25', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2025-04-04', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-12-29', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Symptom Severity', 'timeFrame': '40 min after drug or placebo administration', 'description': 'A combined clinician-objective and patient-subjective change in visual analog scale (min-max 0-100, a higher score is the worse outcome) score of symptom severity before and after drug vs. placebo intake.\n\nFor analysis, patients in groups (1) Alcohol-responsive (EtOH+) Laryngeal Dystonia and (2) Alcohol-responsive (EtOH+) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH+ group.\n\nFor analysis, patients in groups (3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia and (4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH- group.'}, {'measure': 'Minimum Treatment Efficacy', 'timeFrame': '40 min after drug or placebo administration', 'description': 'Pre-specified to analysis based only on alcohol responsiveness (EtOH+). A combined clinician-objective and patient-subjective change in visual analog scale score (min-max 0-100, a higher score is the worse outcome) of symptom severity in EtOH+ patients.'}], 'secondaryOutcomes': [{'measure': 'Treatment Efficacy Dependent on LD Clinical Type', 'timeFrame': '40 min after drug or placebo administration', 'description': 'A combined clinician-objective and patient-subjective change in visual analog scale (min-max 0-100, a higher score is the worse outcome) score of symptom severity before and after drug vs. placebo intake.\n\nEtOH+ and EtOH- were stratified based on phenotypical characteristics, including LD, LD with Dystonic Tremor of Voice, Abductor LD, Adductor LD.'}, {'measure': 'Length of Treatment Efficacy', 'timeFrame': '40 min to 5 hours after drug or placebo administration', 'description': 'The length of treatment efficacy in each EtOH+ and EtOH- group was assessed at 40, 180, and 300 min after drug vs. placebo intake compared to the baseline using a combined clinician-objective and patient-subjective change in visual analog scale (min-max 0-100, a higher score is the worse outcome) score of symptom severity.\n\nFor analysis, patients in groups (1) Alcohol-responsive (EtOH+) Laryngeal Dystonia and (2) Alcohol-responsive (EtOH+) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH+ group.\n\nFor analysis, patients in groups (3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia and (4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH- group.'}, {'measure': 'Relationship Between Alcohol-responsiveness of Symptoms and Drug-induced Symptom Improvement', 'timeFrame': '40 min after drug or placebo administration', 'description': 'A combined clinician-objective and patient-subjective change in visual analog scale (min-max 0-100, a higher score is the worse outcome) score of symptom severity before and 40 min after drug vs. placebo intake.\n\nFor analysis, patients in groups (1) Alcohol-responsive (EtOH+) Laryngeal Dystonia and (2) Alcohol-responsive (EtOH+) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH+ group.\n\nFor analysis, patients in groups (3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia and (4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH- group.'}, {'measure': 'Relationship Between Symptom Severity Change and Dystonia Clinical Characteristics', 'timeFrame': '40 min after drug or placebo administration', 'description': 'A combined clinician-objective and patient-subjective change in visual analog scale (min-max 0-100, a higher score is the worse outcome) score of symptom severity before and 40 min after drug vs. placebo intake.\n\nFor analysis, patients in groups (1) Alcohol-responsive (EtOH+) Laryngeal Dystonia and (2) Alcohol-responsive (EtOH+) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH+ group.\n\nFor analysis, patients in groups (3) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia and (4) Alcohol-non-responsive (EtOH-) Laryngeal Dystonia with Dystonic Tremor of Voice were combined into the overall EtOH- group.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['spasmodic dysphonia', 'laryngeal dystonia', 'functional MRI', 'Xyrem'], 'conditions': ['Spasmodic Dysphonia', 'Voice Tremor']}, 'referencesModule': {'references': [{'pmid': '24386608', 'type': 'BACKGROUND', 'citation': 'Simonyan K, Frucht SJ. Long-term Effect of Sodium Oxybate (Xyrem(R)) in Spasmodic Dysphonia with Vocal Tremor. Tremor Other Hyperkinet Mov (N Y). 2013 Dec 9;3:tre-03-206-4731-1. doi: 10.7916/D8CJ8C5S. eCollection 2013.'}, {'pmid': '27808415', 'type': 'BACKGROUND', 'citation': 'Rumbach AF, Blitzer A, Frucht SJ, Simonyan K. An open-label study of sodium oxybate in Spasmodic dysphonia. Laryngoscope. 2017 Jun;127(6):1402-1407. doi: 10.1002/lary.26381. Epub 2016 Nov 3.'}, {'pmid': '27346568', 'type': 'BACKGROUND', 'citation': 'Battistella G, Fuertinger S, Fleysher L, Ozelius LJ, Simonyan K. Cortical sensorimotor alterations classify clinical phenotype and putative genotype of spasmodic dysphonia. Eur J Neurol. 2016 Oct;23(10):1517-27. doi: 10.1111/ene.13067. Epub 2016 Jun 27.'}, {'pmid': '26843004', 'type': 'BACKGROUND', 'citation': 'Kirke DN, Battistella G, Kumar V, Rubien-Thomas E, Choy M, Rumbach A, Simonyan K. Neural correlates of dystonic tremor: a multimodal study of voice tremor in spasmodic dysphonia. Brain Imaging Behav. 2017 Feb;11(1):166-175. doi: 10.1007/s11682-016-9513-x.'}, {'pmid': '25929664', 'type': 'BACKGROUND', 'citation': 'Kirke DN, Frucht SJ, Simonyan K. Alcohol responsiveness in laryngeal dystonia: a survey study. J Neurol. 2015 Jun;262(6):1548-56. doi: 10.1007/s00415-015-7751-2. Epub 2015 May 1.'}, {'pmid': '20194686', 'type': 'BACKGROUND', 'citation': 'Simonyan K, Ludlow CL. Abnormal activation of the primary somatosensory cortex in spasmodic dysphonia: an fMRI study. Cereb Cortex. 2010 Nov;20(11):2749-59. doi: 10.1093/cercor/bhq023. Epub 2010 Mar 1.'}], 'seeAlsoLinks': [{'url': 'https://reporter.nih.gov/search/dPfQ17fnGUOqdn2Teo9Xwg/project-details/10240318', 'label': 'NIH grant description'}]}, 'descriptionModule': {'briefSummary': 'Using a comprehensive approach of clinico-behavioral testing, neuroimaging and pharmacogenetics, the researchers will examine the clinical effects of sodium oxybate and the matched placebo on voice symptoms in spasmodic dysphonia and voice tremor.', 'detailedDescription': '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. SD becomes even more incapacitating when it is associated with dystonic voice tremor (VT), which is present in about 1/3 of SD patients and is characterized by the inability to sustain a vowel for more than a few seconds. Current treatment of these disorders is limited to the temporary management of voice symptoms with repeated injections of botulinum toxin into the laryngeal muscles. These injections, however, are not fully effective in all SD patients and even less so in combined SD and VT cases. There is, therefore, a critical need to identify alternative therapeutic options that specifically target the pathophysiology of these disorders. On the other hand, the design and the use of such novel therapeutic approaches will be largely unattainable if their central mechanisms of action remain unknown. The objective of this study is to elucidate the primary determinants of clinical response to a novel oral medication, sodium oxybate (Xyrem®), in alcohol-responsive SD and VT patients. Using a comprehensive approach of clinico-behavioral testing, neuroimaging and pharmacogenetics, we aim to determine the clinical response of SD and VT symptoms to sodium oxybate and identify the primary markers of its clinical benefits. This study will use a controlled experimental design that focuses on detailed characterization of primary effects of a novel oral medication, sodium oxybate, for treatment of SD and VT symptoms.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '21 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n1. Patients with SD and combined SD and VT will have a clinically documented adductor or abductor form of disorder, either with or without positive effects of alcohol on their voice symptoms;\n2. Healthy controls will be healthy volunteers with a negative history of laryngeal, neurological, or psychiatric problems (existing neuroimaging data will be used);\n3. Age from 21 to 80 years.\n4. Native English speakers.\n5. Right-handedness (based on Edinburgh Handedness Inventory).\n\nExclusion Criteria:\n\n1. Subjects who are incapable of giving an informed consent will be excluded from the study.\n2. Pregnant and breastfeeding women until a time when they are no longer pregnant or breastfeeding will be excluded from the study. All patients of childbearing potential will be required to agree to use a reliable method of contraception prior to and during the study. The method of contraception will be documented in the patient's research chart. All women of childbearing potential will undergo a urine pregnancy test, which must be negative for study participation.\n3. All patients with a past or present history of the following conditions will be excluded from the study;\n\n 1. Except for SD and dystonic VT, any neurological disorders, such as stroke, movement disorders, brain tumors, traumatic brain injury with loss of consciousness, ataxias, myopathies, myasthenia gravis, demyelinating diseases, alcoholism, drug dependence. Patients with tremor affecting other body parts will be excluded from the study. All patients who have dystonic movements in the body regions other than the larynx will be excluded from the study. This will allow maintaining the homogenous patient population and evaluating central drug effects without confounding by the presence of other neurological conditions.\n 2. Any psychiatric problems, such as schizophrenia, major and/or bipolar depression, obsessive-compulsive disorder, will be excluded to maintain the homogenous patient population and allow for the evaluation of central drug effect without confounding by the presence of psychiatric conditions.\n 3. Any laryngeal problems, such as vocal fold paralysis, paresis, carcinoma, chronic laryngitis, will be excluded from the study.\n 4. Patients with a known past or present history of grade 2 or higher hepatic and renal dysfunction according to the NCI criteria will be excluded.\n 5. Patients with a known past or present history of moderate to severe congestive heart failure will be excluded.\n 6. Patients with a known past or present history of cognitive impairment and active suicidal ideations will be excluded.\n4. Patients who are not symptomatic due to treatment with botulinum toxin injections into the laryngeal muscles will be excluded from the study until the time when they are fully symptomatic. The duration of positive effects of botulinum toxin vary from patient to patient, lasting on average 3-4 months. All patients will be evaluated to ensure that they are fully symptomatic prior to the entering the study.\n5. To avoid the possibility of confounding effects of drugs acting upon the central nervous system, all patients will be questioned about any prescribed or over-the-counter medications as part of their initial intake screening. Those patients who receive medication(s) affecting the central nervous system (except for sodium oxybate) will be excluded from the study.\n6. Patients will be asked whether they have undergone any head and neck surgeries, particularly any brain surgery and laryngeal surgeries, such as thyroplasty, laryngeal denervation, and selective laryngeal adductor denervation-reinnervation. Because both brain and laryngeal surgery may potentially lead to the brain structure and function re-organization, all subjects with a history of brain and/or laryngeal surgery will be excluded from the study.\n7. Patients who have 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 will be excluded from the study."}, 'identificationModule': {'nctId': 'NCT03292458', 'briefTitle': 'Sodium Oxybate in Spasmodic Dysphonia and Voice Tremor', 'organization': {'class': 'OTHER', 'fullName': 'Massachusetts Eye and Ear Infirmary'}, 'officialTitle': 'Central Mechanisms and Treatment Response of Sodium Oxybate in Spasmodic Dysphonia and Voice Tremor', 'orgStudyIdInfo': {'id': '2019P001680'}, 'secondaryIdInfos': [{'id': 'R01DC012545', 'link': 'https://reporter.nih.gov/quickSearch/R01DC012545', 'type': 'NIH'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Clinical response to sodium oxybate', 'description': 'In contrast to placebo but similar to alcohol, sodium oxybate is expected to be most effective in reducing voice symptoms in alcohol-responsive SD and VT.', 'interventionNames': ['Drug: Sodium Oxybate']}, {'type': 'EXPERIMENTAL', 'label': 'Central markers of clinical response', 'description': 'The clinical outcome is expected to be determined by selective modulation of functional abnormalities in the brain regions controlling speech sensorimotor processing and integration in association with underlying gene variants.', 'interventionNames': ['Drug: Sodium Oxybate']}], 'interventions': [{'name': 'Sodium Oxybate', 'type': 'DRUG', 'otherNames': ['Placebo', 'Alcohol'], 'description': 'Alcohol challenge test and oral administration of a single dose of sodium oxybate and the matching placebo.', 'armGroupLabels': ['Central markers of clinical response', 'Clinical response to sodium oxybate']}]}, 'contactsLocationsModule': {'locations': [{'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts Eye and Ear', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}], 'overallOfficials': [{'name': 'Kristina Simonyan, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Massachusetts Eye and Ear'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Kristina Simonyan', 'class': 'OTHER'}, 'collaborators': [{'name': 'National Institute on Deafness and Other Communication Disorders (NIDCD)', 'class': 'NIH'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Professor of Otolaryngology - Head & Neck Surgery, Director of Laryngology Research', 'investigatorFullName': 'Kristina Simonyan', 'investigatorAffiliation': 'Massachusetts Eye and Ear Infirmary'}}}}