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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Clinical response to sodium oxybate (Lumryz) | Experimental | Oral administration of sodium oxybate (1.5g, 2.0g, 2.5g, 3.0g) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| sodium oxybate | Drug | Oral administration of sodium oxybate (1.5g, 2.0g, 2.5g, 3.0g) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy of Lumryz | The primary outcome will be the percent change from baseline in LD symptoms 60 min after each dose of drug intake | 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. | Through study completion, an average of 4 days |
| Measure | Description | Time Frame |
|---|---|---|
| Duration of treatment efficacy | The secondary outcomes will be the duration (in hours) of Lumryz treatment efficacy | Through study completion, an average of 4 days |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Kristina Simonyan, MD, PhD, DrMed | Contact | 6175736025 | kristina_simonyan@meei.harvard.edu |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Massachusetts Eye and Ear | Boston | Massachusetts | 02114 | United States |
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| ID | Term |
|---|---|
| D055154 | Dysphonia |
| ID | Term |
|---|---|
| D014832 | Voice Disorders |
| D007818 | Laryngeal Diseases |
| D012140 | Respiratory Tract Diseases |
| D010038 | Otorhinolaryngologic Diseases |
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| ID | Term |
|---|---|
| D012978 | Sodium Oxybate |
| ID | Term |
|---|---|
| D006885 | Hydroxybutyrates |
| D002087 | Butyrates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
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| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D009930 |
| Organic Chemicals |
| D006880 | Hydroxy Acids |