A Study to Investigate the Effects of Cleminorexton Compared With Placebo in the Treatment of Participants With Central Disorders of Hypersomnolence

Официальное название

A Randomized, Double-blind, 3-Arm Parallel Design Study to Investigate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Cleminorexton Compared With Placebo in Participants With Central Disorders of Hypersomnolence

Обзор

Narcolepsy Type 1 (NT1) and Narcolepsy Type 2 (NT2) are rare conditions that make people feel very sleepy during the day (often referred to as excessive daytime sleepiness \[EDS\]). People living with these conditions might find it hard to stay alert and pay attention when they are at school, working, driving, or performing other daily activities. While all conditions result in feeling sleepy, there are some differences in other common symptoms: * NT1: People with NT1 often feel very tired during the day and experience cataplexy. Cataplexy is a sudden loss of muscle strength, which can cause someone to collapse or lose control of their muscles for a short time. This is often triggered by strong emotions, such as laughter or surprise. They may also have trouble sleeping well at night. * NT2: People with NT2 feel sleepy during the day, just like NT1, but they do not have cataplexy. Orexin is a protein in the brain that helps coordinate a system that plays an important role in helping people to stay awake during the daytime. Cleminorexton is designed to mimic the action of orexin. The purpose of this study is to see how safe and tolerable cleminorexton is in NT1 and NT2 and learn about what the drug does to the body. Another goal of the study is to see if cleminorexton can help people with NT1 and NT2 feel less sleepy and make other symptoms better.

Подробное описание

The SAPPHIRE Clinical Trial

The Sponsor may in the future, through a protocol amendment, expand the protocol to include participants with idiopathic hypersomnia (IH) in Cohort C.

Вмешательства

• Препарат cleminorexton
  Oral cleminorexton capsule
• Препарат Placebo
  matching placebo capsule

Первичные конечные точки

• Change from baseline in mean sleep latency (average of the first 4 trials) on the Maintenance of Wakefulness Test (MWT) [Срок оценки: Baseline to week 12]

Вторичные конечные точки (12)

• Change from baseline in Epworth Sleepiness Scale (ESS) total score [Срок оценки: Baseline to Week 12]
• Weekly Cataplexy Rate (WCR) [Срок оценки: Week 12]
• Change from baseline in Narcolepsy Severity Scale for Clinical Trials (NSS-CT) [Срок оценки: Baseline to Week 12]
• Change from baseline in Narcolepsy Severity Scale-2 for Clinical Trials (NSS2-CT) total score [Срок оценки: Baseline to Week 12]
• Incidence, Severity, and casual relationship of participants with Treatment Emergent Adverse Events (TEAEs) [Срок оценки: Baseline to Week 12]
• Number of participants with abnormal changes from baseline in clinical laboratory tests [Срок оценки: Baseline to Week 12]
• Number of participants with abnormal changes from baseline in vital signs [Срок оценки: Baseline to Week 12]
• Number of participants with abnormal changes from baseline in 12-lead electrocardiograms (ECGs) [Срок оценки: Baseline to Week 12]
• Number of participants with at least 1 endorsement of Item 4 or 5 on Columbia-Suicide Severity Rating Scale (C-SSRS) [Срок оценки: Baseline to Week 12]
• Cmax: Maximum observed plasma concentration [Срок оценки: Baseline to Week 12]
• Tmax: Time of Maximum Concentration for cleminorexton [Срок оценки: Baseline to Week 12]
• AUC: Area under the plasma concentration-time curve [Срок оценки: Baseline to Week 12]

Критерии участия

Критерии включения

• 18-70 years of age
• Body Mass Index (BMI) within the range ≥ 17.0 and ≤ 45 kg/m\^2 (inclusive)
• Meets the diagnostic criteria of Narcolepsy Type 1 (NT1) or Type 2 (NT2) according to International Classification of Sleep Disorders, 3rd edition, Text Revision edition (ICSD-3-TR) criteria
• Is willing and able to discontinue all medications used for the treatment of narcolepsy
• Is willing and able to adhere to additional protocol requirements

Критерии исключения

• Medical disorder other than NT1 or NT2, that is associated with excessive daytime sleepiness (EDS)
• Presence of significant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, hematological, malignancy, endocrine, neurological or psychiatric disease

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 10 центров

• Colorado Springs, Colorado — Colorado Springs
• Miami, Florida — Miami
• Miami Lakes, Florida — Miami Lakes
• Flowood Mississippi — Flowood
• Denver, North Carolina — Denver
• Huntersville, North Carolina — Huntersville
• Cincinnati, Ohio — Cincinnati
• Dublin, Ohio — Dublin
• … и ещё 2 центра

Идентификаторы

NCT: NCT07598708 · ORX750-004

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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