Extended-release Sodium Oxybate in Children

Официальное название

Safety, Convenience, and Tolerability of a Nightly Single Oral Dose of Extended-release Sodium Oxybate in Children

Обзор

The main objectives of this study are to determine the treatment preferences of children with narcolepsy type 1 between Extended-release sodium oxybate and Non-extended-release oxybates (Sodium oxybate or a medication with the combination of Calcium, magnesium, potassium, and sodium oxybates). The study will also assess the safety, convenience, and tolerability of Extended-release sodium oxybate. Additionally, the researchers aim to assess how well Extended-release sodium oxybate works in treating narcolepsy type 1.

Вмешательства

• Препарат Extended-release sodium oxybate
  Participants will take Extended-release sodium Oxybate (Lumryz) 4.5-9 g oral suspension in approximately ⅓ cup of water orally once a day before bedtime. Dosage of Lumryz will be the same as the equivalent total dose of the Non-Extended-release Oxybate (Xywav or Xyrem) that participants use before starting the study
• Препарат Non-extended-release oxybates
  Participants will take Non-Extended-release Oxybates (Xyrem oral solution or Xywav oral solution) 4.5-9 g orally per night (divided into two doses, once before bedtime and another dose 2-4 hours later) at the same dose of Xyrem or Xywav that participants use before starting the study will be continued to compare with Extended-release sodium Oxybate (Lumryz)

Первичные конечные точки

• Participant preference for selecting the extended vs non-extended release oxybates [Срок оценки: Baseline, up to 8-12 weeks through study completion]

Вторичные конечные точки (3)

• Change in Epworth Sleepiness Scales for Children and Adolescents (ESS-CHAD) score [Срок оценки: Baseline, up to 8-12 weeks through study completion]
• Number of cataplexy attacks [Срок оценки: Baseline, up to 8-12 weeks through study completion]
• Change in Patient Global Impression of Change (PGIc) scale score [Срок оценки: Baseline, up to 8-12 weeks through study completion]

Критерии участия

Критерии включения

• Participants must be under the care of a doctor at the Stanford Sleep Clinic.
• Participants must have a documented diagnosis of narcolepsy type 1 shown by sleepiness and either: a spinal fluid marker for narcolepsy (hypocretin-1) at a specific level, or a history of sudden loss of muscle control (cataplexy), or a particular genetic marker for narcolepsy, or a sleep study showing a specific sleep pattern for narcolepsy.
• Parent(s), or guardian(s) have signed a consent form and the child must agree to participate.
• Participants are on a stable dose of medications

Критерии исключения

Participants who have any of the following conditions will not be included in the study

• Uncontrolled mental health problems
• Uncontrolled sleep problems that lead to sleepiness.
• Currently having thought about ending one's life or sadness or loss of interest
• Currently having a problem with illegal drug use
• Currently pregnant

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Перекрёстный дизайн

Маскирование

Открытое

Основная цель

Другое

Центры проведения

США · 1 центр

• Stanford University — Redwood City

Идентификаторы

NCT: NCT06809803 · 75896

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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