Rare Neurology News
Spotlight On
Narcolepsy
Narcolepsy is a chronic brain disorder that involves poor control of sleep-wake cycles. People with narcolepsy have episodes of extreme daytime sleepiness and sudden, irresistible bouts of sleep (called "sleep attacks") that can occur at any time, and may last from seconds or minutes
Prevelance
1-5 / 10,000
Age of Onset
ICD-10
G47.4
Inheritance
Autosomal dominant
Autosomal recessive
Mitochondrial/Multigenic
X-linked dominant
X-linked recessive
Rare View
Narcolepsy is a disorder that disrupts sleep-wake processes. Its primary symptom is excessive daytime sleepiness (EDS), which occurs because the brain is unable to properly regulate wakefulness and sleep. The cause of narcolepsy isn't well understood but may involve genetic factors and abnormal signaling in the brain. Narcolepsy causes sudden attacks of sleep. Sudden loss of muscle tone and hallucinations might occur.†
5 Facts you should know
FACT
The most typical symptoms are excessive daytime sleepiness, cataplexy, sleep paralysis, and hallucinations
FACT
About 70% of those affected also experience episodes of sudden loss of muscle strength, known as cataplexy
FACT
There is increasing evidence that narcolepsy is an autoimmune disorder
FACT
Type 1 narcolepsy is defined as narcolepsy with cataplexy; Type 2 is narcolepsy without cataplexy
FACT
The loss of hypocretin-producing neurons in the hypothalamus is the primary cause of type 1 narcolepsy
Interest over time
Google searches
Common signs & symptoms
Cataplexy
Excessive daytime somnolence
Excessive daytime sleepiness
Hallucinations
Sensory hallucination
Transient global amnesia
Abnormal rapid eye movement sleep
Abnormality of vision
Abnormality of sight
Slurred speech
Syncope
Fainting spell
Current treatments
The medication(s) listed below have been approved by the Food and Drug Administration (FDA) as orphan products for treatment of this condition. Learn more orphan products.
Modafinil
(Brand name: Provigil) Manufactured by Cephalon, Inc.
FDA-approved indication: Improve wakefulness in patients with excessive daytime sleepiness associated with narcolepsy.
Oxybate
(Brand name: Xyrem®) Manufactured by Jazz Pharmaceuticals
FDA-approved indication: Treatment of cataplexy associated with narcolepsy.
Top Clinical Trials
| Title | Description | Phases | Status | Interventions | More Information |
|---|---|---|---|---|---|
| Modafinil Versus Amphetamines for the Treatment of Narcolepsy Type 2 and Idiopathic Hypersomnia | This study will evaluate which medication works better to improve sleepiness. The researchers will also see which medication is better for other symptoms including difficulty waking up and difficulty thinking, as well as seeing which medication causes fewer side. Finally, this study will see if any information about patients (such as age or sleep study features) predicts responding better to one medication or the other. | Phase 2 | Recruiting | Drug: Modafinil|Drug: Amphetamine-Dextroamphetamine | Click here for more information |
| Study to Evaluate the Abuse Liability, Pharmacokinetics, Safety and Tolerability of an Abuse-Deterrent d-Amphetamine Sulfate Immediate Release Formulation (ADAIR) | This is a randomized, double-blind, double-dummy, placebo- and active-controlled 4 period, 4 way crossover study to assess the intranasal abuse potential of manipulated ADAIR formulation in nondependent, recreational stimulant users. The study will consist of an outpatient Screening Visit, an in clinic Qualification Phase, an in-clinic Treatment Phase, and an outpatient Follow-Up visit. | Phase 1 | Recruiting | Drug: ADAIR 10 mg IR tablets|Drug: d-amphetamine sulfate|Drug: Placebo | Click here for more information |
| Clarithromycin Mechanisms in Hypersomnia Syndromes | The purpose of this study is to evaluate a medication called clarithromycin for treating sleepiness in two related conditions, narcolepsy without cataplexy and idiopathic hypersomnia. Studies have shown that clarithromycin can reduce sleepiness, but researchers do not know how clarithromycin does this. This study will look at brain activity (on magnetic resonance imaging [MRI] and electroencephalogram [EEG] brainwaves), inflammation, bacteria living in the gut, and cerebrospinal fluid, to better understand how clarithromycin can reduce sleepiness. This study will recruit 92 participants who will be randomized to receive clarithromycin or a placebo for 14 days. | Phase 2 | Recruiting | Drug: Clarithromycin|Drug: Placebo | Click here for more information |
| A Study to Evaluate Safety, and Efficacy of SUVN-G3031 in Patients With Narcolepsy With and Without Cataplexy | This study is of an investigational drug called SUVN-G3031 as a possible treatment for narcolepsy with cataplexy or narcolepsy without cataplexy. The main purpose of this study is to learn how well the study drug works and how safe the study drug is compared to placebo. | Phase 2 | Recruiting | Drug: SUVN-G3031|Drug: Placebo | Click here for more information |
| A Study of TAK-994 in Adults With Type 1 and Type 2 Narcolepsy | The main aims of the study are: To check for side effects from TAK-994 and check what dose of TAK-994 participants can tolerate. To check what dose range provides adequate relief of narcolepsy symptoms. To check how much TAK-994 stays in the blood of participants, over time. | Phase 2 | Recruiting | Drug: TAK-994|Drug: Placebo | Click here for more information |
| An Open Label Study of FT218 in Subjects With Narcolepsy | An Open Label Study to Evaluate Long-Term Safety and Tolerability of a Once Nightly Formulation of Sodium Oxybate for Extended-Release Oral Suspension (FT218) and the ability to switch from twice-nightly immediate release sodium oxybate to once-nightly FT218 for the Treatment of Excessive Daytime Sleepiness and Cataplexy in Subjects with Narcolepsy | Phase 3 | Recruiting | Drug: FT218 | Click here for more information |
Top Treatments in Research
| Agent | Class/Mechanism of Action | Development Status | Company | Clinical Studies | More Information |
|---|---|---|---|---|---|
| TAK-994 | Oral orexin agonist - novel, orally available OX2R-selective agonist with >700-fold selectivity against orexin 1 receptor. | Phase II | Takeda Pharmaceutical Company | Click here for more information | Click here for more information |
| FT218 | Extended-release formulation of sodium oxybate | Phase III | Avadel Pharmaceuticals | Click here for more information | Click here for more information |
| SUVN-G3031 | Orally-bioavailable Histamine H3 Receptor Inverse Agonist | Phase II | Suven Life Sciences Limited | Click here for more information | Click here for more information |
| AXS-12 (reboxetine) | Selective norepinephrine reuptake inhibitor thought to modulate noradrenergic activity to promote wakefulness, maintain muscle tone and enhance cognition | Phase II | Axsome Therapeutics | Click here for more information | Click here for more information |
† Genetic and Rare Diseases Information Center (GARD) - PO Box 8126, Gaithersburg, MD 20898-8126 https://rarediseases.info.nih.gov