Narcolepsy without cataplexy

Summary

Narcolepsy without cataplexy (MONDO:0019371) is a disease and 24 clinical trials. Top therapeutic interventions include pitolisant, flumazenil, and mazindol. A subtype of brain disorder — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

• Prevalence: 1-5 / 10 000 (United States) [Orphanet-validated]
• Phenotypes (HPO): 5
• Clinical trials: 24

Clinical features

Epidemiology

Prevalence records

1 prevalence record(s), Orphanet:

Type	Class	Value	Geography	Validation
Point prevalence	1-5 / 10 000	20.3	United States	Validated

Signs & symptoms

Clinical features (HPO)

5 HPO clinical features (Orphanet curated; top 5 by frequency):

HPO ID	Term	Frequency
HP:0000738	Hallucinations	Very frequent (80-99%)
HP:0001262	Excessive daytime somnolence	Very frequent (80-99%)
HP:0002360	Sleep abnormality	Very frequent (80-99%)
HP:0100785	Insomnia	Very frequent (80-99%)
HP:0000708	Atypical behavior	Occasional (5-29%)

Identifiers

Disease identifiers

Field	Value
Canonical name	narcolepsy without cataplexy
Mondo ID	MONDO:0019371
EFO	EFO:0005855
Orphanet	83465
ICD-10-CM	G47.419
ICD-11	1494673323
SNOMED CT	91521000119104
UMLS	C1456240
MedGen	781102
GARD	0019038
Is cancer (heuristic)	no

Also known as: narcolepsy type 2

Disease family

This is a subtype of brain disorder. Genetic, therapeutic, and trial evidence is largely curated at the broader-term level — see the parent page for the associated-gene cohort and molecular evidence.

Classification path: disease › human disease › disease by body system or component › nervous system disorder › central nervous system disorder › brain disorder › narcolepsy without cataplexy

Related subtypes (70): leukoencephalopathy, megalencephalic, encephalopathy, acute, infection-induced, diabetic encephalopathy, complex cortical dysplasia with other brain malformations, hydrocephalus, brain compression, cerebral sarcoidosis, hepatic encephalopathy, visual pathway disorder, central nervous system origin vertigo, cerebellar disorder, cerebritis, olfactory nerve disorder, thalamic disorder, pituitary gland disorder, disorder of optic chiasm, basal ganglia disorder, epilepsy, mental disorder, central nervous system cyst, migraine disorder, multiple sclerosis, prion disease, carbon monoxide-induced delayed encephalopathy, cerebral malaria, akinetic mutism, bulbar polio, Reye syndrome, brain edema, encephalomalacia, intracranial hypertension, intracranial hypotension, Wernicke encephalopathy, encephalopathy, recurrent, of childhood, XK aprosencephaly, progressive bulbar palsy, cerebrovascular disorder, glycine encephalopathy, autosomal recessive frontotemporal pachygyria, occipital pachygyria and polymicrogyria, insomnia, narcolepsy-cataplexy syndrome, megalencephaly, meningoencephalocele, cerebral cortical dysplasia, encephaloclastic disorder, bilirubin encephalopathy, autoimmune encephalopathy with parasomnia and obstructive sleep apnea, hypothalamic hamartomas with gelastic seizures, encephalitis, cerebral lipidosis with dementia, brain neoplasm, colpocephaly, corpus callosum agenesis of blepharophimosis robin type, corpus callosum dysgenesis X-linked recessive, corpus callosum dysgenesis cleft spasm, corpus callosum dysgenesis hypopituitarism, cerebral degeneration, acute bilirubin encephalopathy, chronic bilirubin encephalopathy, atelencephaly, aprosencephaly, brain injury, traumatic encephalopathy, cluster headache syndrome, cerebral cortex disorder, midbrain disorder, encephalopathy due to mitochondrial and peroxisomal fission defect, brain malformations with or without urinary tract defects, encephalopathy, acute transient

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

No tiered GWAS variants or ClinVar records for this disease.

Genes & proteins

No associated-gene cohort resolved for this disease. Atlas builds the molecular and therapeutic sections — associated genes, protein families, druggability, pathways, interactions, and drug associations — by aggregating over a disease’s associated genes (resolved via GWAS / GenCC / ClinVar / CIViC), and none resolved here. This is expected for antibody-mediated, autoimmune, or otherwise non-gene-defined conditions; the curated evidence for this disease is its clinical features, GWAS susceptibility, and clinical trials (above).

Function

No pathway enrichment — requires an associated-gene cohort.

Therapeutics

No druggable-target or therapeutic data for this disease’s cohort.

Clinical trials & evidence

Clinical trials

Clinical trials: 24.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	10
Not specified	9
PHASE3	2
PHASE2/PHASE3	2
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06767683	PHASE2/PHASE3	RECRUITING	A Long-Term Study of ALKS 2680 in Subjects With Narcolepsy and Idiopathic Hypersomnia
NCT07502443	PHASE3	RECRUITING	A Study to Evaluate the Efficacy and Safety of ALKS 2680 in Adults With Narcolepsy Type 2
NCT07598708	PHASE2/PHASE3	NOT_YET_RECRUITING	A Study to Investigate the Effects of Cleminorexton Compared With Placebo in the Treatment of Participants With Central Disorders of Hypersomnolence
NCT02611687	PHASE3	COMPLETED	Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period
NCT06752668	PHASE2	RECRUITING	A Study of ORX750 in Participants With Narcolepsy and Idiopathic Hypersomnia
NCT06952699	PHASE2	RECRUITING	A Study of TAK-360 in Adults With Narcolepsy Without Cataplexy (NT2)
NCT07096674	PHASE2	RECRUITING	A Long-term Extension Study of ORX750 in Participants With Narcolepsy and Idiopathic Hypersomnia
NCT01183312	PHASE1/PHASE2	COMPLETED	Flumazenil for the Treatment of Primary Hypersomnia
NCT02512588	PHASE2	COMPLETED	A Study of Safety and Efficacy of BTD-001 in Treatment of Patients With Idiopathic Hypersomnia (IH) or Narcolepsy Type 2
NCT03772314	PHASE2	COMPLETED	Modafinil Versus Amphetamines for the Treatment of Narcolepsy Type 2 and Idiopathic Hypersomnia
NCT04026958	PHASE2	COMPLETED	Clarithromycin Mechanisms in Hypersomnia Syndromes
NCT04096560	PHASE2	TERMINATED	A Study of TAK-994 in Adults With Type 1 and Type 2 Narcolepsy
NCT05055024	PHASE2	COMPLETED	An Open Label Study of NLS-2 (Mazindol Extended Release) in Subjects With Narcolepsy
NCT05687916	PHASE2	COMPLETED	A Study of TAK-861 in Participants With Narcolepsy Type 2
NCT06555783	PHASE2	COMPLETED	A Study to Evaluate the Safety and Effectiveness of ALKS 2680 in Subjects With Narcolepsy Type 2
NCT01793168	Not specified	RECRUITING	Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
NCT06383806	Not specified	NOT_YET_RECRUITING	Decreasing Nightmares in Adults With Narcolepsy
NCT07006233	Not specified	RECRUITING	A Novel Approach to Manage Symptoms of Narcolepsy and Idiopathic Hypersomnia
NCT07299097	Not specified	RECRUITING	Epidemiology of Narcolepsy Type 1 and Type 2 in Spain
NCT03433131	Not specified	NO_LONGER_AVAILABLE	Expanded Access Program to Provide Treatment With Pitolisant to Adult Patients in the U.S. With Excessive Daytime Sleepiness Associated With Narcolepsy With or Without Cataplexy
NCT04306952	Not specified	COMPLETED	Awareness and Self-Compassion Enhancing Narcolepsy Treatment
NCT04419792	Not specified	SUSPENDED	‘A Profile of Physical Performance Variables in an Out-patient Adult Population With Narcolepsy’
NCT05709873	Not specified	COMPLETED	Narcolepsy Nightmare Study
NCT06251063	Not specified	COMPLETED	Improving Social Relationships for Adolescents With Central Disorders of Hypersomnolence

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
PITOLISANT	4	6
FLUMAZENIL	4	1
MAZINDOL	4	1
MODAFINIL	4	1