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Набор скоро начнётся NCT07041203

Extended-release Sodium Oxybate (Lumryz) in Spasmodic Dysphonia and Voice Tremor

Фаза I С лечением Spasmodic Dysphonia Laryngeal Dystonia Voice Tremor

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают
В протоколе указаны: sodium oxybate.
Кому может быть актуально
Состояния в реестре: Spasmodic Dysphonia, Laryngeal Dystonia, Voice Tremor. Базовые параметры: 21 лет — 80 лет · Все.
Что важно проверить
Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится
США
Следующий шаг
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Официальное название

Central Mechanisms and Treatment Response of Sodium Oxybate (Lumryz) in Spasmodic Dysphonia and Voice Tremor

Обзор

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.

Подробное описание

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.

Вмешательства

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

Первичные конечные точки

  • Efficacy of Lumryz [Срок оценки: Through study completion, an average of 4 days]
  • Efficacy of Lumryz [Срок оценки: Through study completion, an average of 4 days]
Вторичные конечные точки (1)
  • Duration of treatment efficacy [Срок оценки: Through study completion, an average of 4 days]

Критерии участия

Критерии включения

  • Provision of signed and dated informed consent form
  • Stated willingness to comply with all study procedures and lifestyle considerations (see Lifestyle Considerations below) and availability for the duration of the study
  • Males and females
  • Age 21-80 years
  • Documented diagnosis of alcohol-responsive laryngeal dystonia
  • Documented positive response to immediate-release sodium oxybate (Xyrem) in prior studies
  • Willingness to adhere to the study intervention regimen

Критерии исключения

  • The incapability of giving informed consent
  • Pregnancy or breastfeeding until a time when they are no longer pregnant or breastfeeding
  • Grade 2 or higher hepatic or renal dysfunction according to the NCI criteria
  • Moderate to severe congestive heart failure
  • Cognitive impairment (MoCA < 26)
  • Past or present suicidal ideations (according to C-SSRS)
  • Alcoholism or high risk for alcohol use disorder according to the NIAAA definition and DSM-5 criteria
  • Asymptomatic presentation due to botulinum toxin treatment until the time they are fully symptomatic and are at least 3 months after the last injection
  • Increased daytime sleepiness (Epworth Sleepiness Scale (ESS>10))
  • 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.
  • Past or present history of any psychiatric problems, such as schizophrenia, major and/or bipolar depression, or obsessive-compulsive disorder
  • Current use of medication(s) affecting the central nervous system
  • Past or present history of brain and/or laryngeal surgery
  • 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение
Не применимо
Модель
Одна группа
Маскирование
Открытое
Основная цель
Лечение

Центры проведения

США · 1 центр
  • Massachusetts Eye and Ear — Boston

Идентификаторы

NCT: NCT07041203 · 2025P000919

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗