A Study of ORX750 in Participants With Narcolepsy and Idiopathic Hypersomnia

Официальное название

A Phase 2a, Randomized, Double-blind, Placebo-controlled Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ORX750 in Subjects With Narcolepsy and Idiopathic Hypersomnia (CRYSTAL-1)

Обзор

Narcolepsy Type 1 (NT1), Narcolepsy Type 2 (NT2), and Idiopathic Hypersomnia (IH) 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. * IH: People with IH feel tired during the day, even after sleeping a lot at night. They may sleep for long periods, take long naps, and find it hard to wake up. 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 (also known as ORX750) is designed to mimic the action of orexin. The purpose of this study is to see how safe and tolerable ORX750 is in NT1, NT2, and IH, and learn about what the drug does to the body. Another goal of the study is to see if ORX750 can help people with NT1, NT2, and IH feel less sleepy and make other symptoms better.

Вмешательства

• Препарат ORX750
  ORX750 capsule.
• Препарат Placebo
  ORX750 matching placebo capsule.

Первичные конечные точки

• Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs [Срок оценки: Up to Day 35]
• Number of Participants With Abnormal Changes From Baseline in Laboratory Tests [Срок оценки: Up to Day 35]
• Number of Participants With Abnormal Change From Baseline in Vital Signs [Срок оценки: Up to Day 35]
• Number of Participants With Abnormal Change From Baseline in Electrocardiograms (ECG) [Срок оценки: Up to Day 35]
• Number of Participants With Suicidal Ideation or Suicidal Behavior as Measured Using Columbia-Suicide Severity Rating Scale (C-SSRS) [Срок оценки: Up to Day 35]

Вторичные конечные точки (7)

• Cmax: Maximum Observed Plasma Concentration for ORX750 [Срок оценки: Day 1, 15, 28]
• Tmax: Time of Maximum Concentration for ORX750 [Срок оценки: Day 1, 15, 28]
• AUC0last: Area Under the Plasma Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration for ORX750 [Срок оценки: up to Day 28]
• Cmax,ss: Maximum Observed Concentration at Steady State for ORX750 [Срок оценки: Day 1, 15, 28]
• AUCτ: Area Under the Concentration-time Curve During a Dosing Interval at Steady State for ORX750 [Срок оценки: Day 1, 15, 28]
• Mean sleep latency (average of the first 4 trials) in the Maintenance of Wakefulness Test (MWT) for ORX750 versus placebo. [Срок оценки: Baseline up to Day 28]
• Change From Baseline in Epworth Sleepiness Scale (ESS) [Срок оценки: Baseline up to Day 28]

Критерии участия

Критерии включения

• 18-65 years of age
• BMI ≥17 and ≤37 kg/m2
• Meets the diagnostic criteria of Narcolepsy Type 1 (NT1), Type 2 (NT2) or Idiopathic Hypersomnia (IH) according to ICSD-3-TR criteria
• Is willing and able to discontinue all medications used for the treatment of narcolepsy or idiopathic hypersomnia
• Is willing and able to adhere to additional protocol requirements

Критерии исключения

• A medical disorder other than NT1, NT2, or IH 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 · 29 центров

• Auburn, Alabama — Auburn
• Chandler, Arizona — Chandler
• Scottsdale, Arizona — Scottsdale
• Long Beach, California — Long Beach
• Santa Ana, California — Santa Ana
• Brandon, Florida — Brandon
• Miami, Florida — Miami
• Miami, Florida — Miami
• … и ещё 21 центр

Италия · 3 центра

• Bologna, Italy — Bologna
• Pozzilli, Italy — Pozzilli
• Verona, Italy — Verona

Испания · 3 центра

• Madrid, Spain — Madrid
• Madrid, Spain — Madrid
• Vitoria-Gasteiz, Spain — Vitoria-Gasteiz

Канада · 1 центр

• Toronto, Ontario — Toronto

Франция · 1 центр

• Leon, France — Léon

Идентификаторы

NCT: NCT06752668 · ORX750-0201 · 2024-518929-15-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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