Ignite Creation Date:
2025-12-24 @ 7:23 PM
Ignite Modification Date:
2025-12-28 @ 10:13 PM
Study NCT ID:
NCT07041203
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-11-26
First Post:
2025-06-04
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Extended-release Sodium Oxybate (Lumryz) in Spasmodic Dysphonia and Voice Tremor
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, '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'}], '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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 8}}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2026-03-01', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2027-07-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-24', 'studyFirstSubmitDate': '2025-06-04', 'studyFirstSubmitQcDate': '2025-06-18', 'lastUpdatePostDateStruct': {'date': '2025-11-26', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-06-27', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-07-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Efficacy of Lumryz', 'timeFrame': 'Through study completion, an average of 4 days', 'description': 'The primary outcome will be the percent change from baseline in LD symptoms 60 min after each dose of drug intake'}, {'measure': 'Efficacy of Lumryz', 'timeFrame': 'Through study completion, an average of 4 days', 'description': 'The primary outcome will be the number of patients (N, % of total) with improved voice symptoms from baseline after each dose of the drug.'}], 'secondaryOutcomes': [{'measure': 'Duration of treatment efficacy', 'timeFrame': 'Through study completion, an average of 4 days', 'description': 'The secondary outcomes will be the duration (in hours) of Lumryz treatment efficacy'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Spasmodic Dysphonia', 'Laryngeal Dystonia', 'Voice Tremor']}, 'descriptionModule': {'briefSummary': '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.', '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. 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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '21 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Provision of signed and dated informed consent form\n2. Stated willingness to comply with all study procedures and lifestyle considerations (see Lifestyle Considerations below) and availability for the duration of the study\n3. Males and females\n4. Age 21-80 years\n5. Documented diagnosis of alcohol-responsive laryngeal dystonia\n6. Documented positive response to immediate-release sodium oxybate (Xyrem) in prior studies\n7. Willingness to adhere to the study intervention regimen\n\nExclusion Criteria:\n\n1. The incapability of giving informed consent\n2. Pregnancy or breastfeeding until a time when they are no longer pregnant or breastfeeding\n3. Grade 2 or higher hepatic or renal dysfunction according to the NCI criteria\n4. Moderate to severe congestive heart failure\n5. Cognitive impairment (MoCA \\< 26)\n6. Past or present suicidal ideations (according to C-SSRS)\n7. Alcoholism or high risk for alcohol use disorder according to the NIAAA definition and DSM-5 criteria\n8. Asymptomatic presentation due to botulinum toxin treatment until the time they are fully symptomatic and are at least 3 months after the last injection\n9. Increased daytime sleepiness (Epworth Sleepiness Scale (ESS\\>10))\n10. 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.\n11. Past or present history of any psychiatric problems, such as schizophrenia, major and/or bipolar depression, or obsessive-compulsive disorder\n12. Current use of medication(s) affecting the central nervous system\n13. Past or present history of brain and/or laryngeal surgery\n14. 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'}, 'identificationModule': {'nctId': 'NCT07041203', 'briefTitle': 'Extended-release Sodium Oxybate (Lumryz) in Spasmodic Dysphonia and Voice Tremor', 'organization': {'class': 'OTHER', 'fullName': 'Massachusetts Eye and Ear Infirmary'}, 'officialTitle': 'Central Mechanisms and Treatment Response of Sodium Oxybate (Lumryz) in Spasmodic Dysphonia and Voice Tremor', 'orgStudyIdInfo': {'id': '2025P000919'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Clinical response to sodium oxybate (Lumryz)', 'description': 'Oral administration of sodium oxybate (1.5g, 2.0g, 2.5g, 3.0g)', 'interventionNames': ['Drug: sodium oxybate']}], 'interventions': [{'name': 'sodium oxybate', 'type': 'DRUG', 'otherNames': ['Lumryz'], 'description': 'Oral administration of sodium oxybate (1.5g, 2.0g, 2.5g, 3.0g)', 'armGroupLabels': ['Clinical response to sodium oxybate (Lumryz)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'contacts': [{'name': 'Kristina Simonyan, MD, PhD, DrMed', 'role': 'CONTACT', 'email': 'kristina_simonyan@meei.harvard.edu', 'phone': '6175736016'}, {'name': 'Kristina Simonyan, MD, PhD, DrMed', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Massachusetts Eye and Ear', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}], 'centralContacts': [{'name': 'Kristina Simonyan, MD, PhD, DrMed', 'role': 'CONTACT', 'email': 'kristina_simonyan@meei.harvard.edu', 'phone': '6175736025'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Kristina Simonyan', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Professor of Otolaryngology-Head and Neck Surgery', 'investigatorFullName': 'Kristina Simonyan', 'investigatorAffiliation': 'Massachusetts Eye and Ear Infirmary'}}}}