A Study of ORX750 in Participants With Narcolepsy and Idiopathic Hypersomnia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ORX750, Placebo.
- Who it may be relevant to
- Registry conditions: Narcolepsy Type 1, Narcolepsy Type 2, Idiopathic Hypersomnia. Basic parameters: 18 years — 65 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Canada, France, Italy, Spain
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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)
Overview
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.
Interventions
- Drug ORX750
ORX750 capsule. - Drug Placebo
ORX750 matching placebo capsule.
Primary outcome measures
- Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious TEAEs [Time frame: Up to Day 35]
- Number of Participants With Abnormal Changes From Baseline in Laboratory Tests [Time frame: Up to Day 35]
- Number of Participants With Abnormal Change From Baseline in Vital Signs [Time frame: Up to Day 35]
- Number of Participants With Abnormal Change From Baseline in Electrocardiograms (ECG) [Time frame: Up to Day 35]
- Number of Participants With Suicidal Ideation or Suicidal Behavior as Measured Using Columbia-Suicide Severity Rating Scale (C-SSRS) [Time frame: Up to Day 35]
Secondary outcome measures (7)
- Cmax: Maximum Observed Plasma Concentration for ORX750 [Time frame: Day 1, 15, 28]
- Tmax: Time of Maximum Concentration for ORX750 [Time frame: 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 [Time frame: up to Day 28]
- Cmax,ss: Maximum Observed Concentration at Steady State for ORX750 [Time frame: Day 1, 15, 28]
- AUCτ: Area Under the Concentration-time Curve During a Dosing Interval at Steady State for ORX750 [Time frame: Day 1, 15, 28]
- Mean sleep latency (average of the first 4 trials) in the Maintenance of Wakefulness Test (MWT) for ORX750 versus placebo. [Time frame: Baseline up to Day 28]
- Change From Baseline in Epworth Sleepiness Scale (ESS) [Time frame: Baseline up to Day 28]
Eligibility criteria
Inclusion criteria
- 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
Exclusion criteria
- 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 29 centers
- 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
- … and 21 more centers
Italy · 3 centers
- Bologna, Italy — Bologna
- Pozzilli, Italy — Pozzilli
- Verona, Italy — Verona
Spain · 3 centers
- Madrid, Spain — Madrid
- Madrid, Spain — Madrid
- Vitoria-Gasteiz, Spain — Vitoria-Gasteiz
Canada · 1 center
- Toronto, Ontario — Toronto
France · 1 center
- Leon, France — Léon
Identifiers
NCT: NCT06752668 · ORX750-0201 · 2024-518929-15-00