About Narcolepsy with Cataplexy
Type 1 narcolepsy (narcolepsy with cataplexy) is caused by autoimmune destruction of hypothalamic hypocretin (orexin)-producing neurons, resulting in CSF hypocretin-1 levels below 110 pg/mL. Clinical tetrad includes excessive daytime sleepiness, cataplexy (sudden loss of muscle tone triggered by emotion), sleep paralysis, and hypnagogic hallucinations. Approved treatments include sodium oxybate (Xyrem/Lumryz), pitolisant (Wakix), and modafinil/solriamfetol for sleepiness.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Narcolepsy with Cataplexy trials.
CSF hypocretin-1 level below 110 pg/mL is the gold standard diagnostic marker for NT1 trials and confirms autoimmune subtype
Polysomnography followed by Multiple Sleep Latency Test (MSLT) showing mean sleep latency <8 min and 2+ SOREMPs is required
Current medications (sodium oxybate, stimulants) require washout periods before PSG/MSLT and before enrollment in many trials
HLA-DQB1*06:02 typing is often performed at screening — presence supports NT1 diagnosis but absence does not exclude
Patient Resources
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