{"id":"double-blind-pitolisant","safety":{"commonSideEffects":[{"rate":null,"effect":"Insomnia"},{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Anxiety"},{"rate":null,"effect":"Nausea"},{"rate":null,"effect":"Irritability"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Pitolisant works by antagonizing H3 receptors on histaminergic neurons, which normally provide negative feedback to suppress histamine release. By blocking this inhibition, the drug enhances histamine-mediated wakefulness promotion. This mechanism increases arousal and reduces excessive daytime sleepiness in conditions characterized by hypersomnia.","oneSentence":"Pitolisant is a selective histamine H3 receptor antagonist that increases wakefulness by blocking inhibitory histamine signaling in the brain.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T01:09:54.137Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Narcolepsy type 1 and type 2"},{"name":"Idiopathic hypersomnia"}]},"trialDetails":[{"nctId":"NCT06366464","phase":"PHASE3","title":"A Study of Pitolisant in Patients With Prader-Willi Syndrome","status":"RECRUITING","sponsor":"Harmony Biosciences Management, Inc.","startDate":"2024-05-28","conditions":"Prader-Willi Syndrome","enrollment":134},{"nctId":"NCT04257929","phase":"PHASE2","title":"A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension","status":"ACTIVE_NOT_RECRUITING","sponsor":"Harmony Biosciences Management, Inc.","startDate":"2020-12-09","conditions":"Prader-Willi Syndrome","enrollment":65},{"nctId":"NCT05953389","phase":"PHASE2","title":"Proof of Concept Study on Pitolisant Effect on Autism Spectrum Disorders in Children and Adolescents","status":"ACTIVE_NOT_RECRUITING","sponsor":"Bioprojet","startDate":"2023-12-20","conditions":"Autism Spectrum Disorder","enrollment":62},{"nctId":"NCT02611687","phase":"PHASE3","title":"Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period","status":"COMPLETED","sponsor":"Bioprojet","startDate":"2016-06-06","conditions":"Narcolepsy With Cataplexy, Narcolepsy Without Cataplexy","enrollment":110},{"nctId":"NCT04886518","phase":"PHASE2","title":"Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1","status":"COMPLETED","sponsor":"Harmony Biosciences Management, Inc.","startDate":"2021-06-28","conditions":"Myotonic Dystrophy 1, Excessive Daytime Sleepiness","enrollment":30},{"nctId":"NCT05156047","phase":"PHASE3","title":"A Phase 3 Study to Assess the Safety and Efficacy of Pitolisant in Adult Patients With Idiopathic Hypersomnia","status":"COMPLETED","sponsor":"Harmony Biosciences Management, Inc.","startDate":"2022-05-25","conditions":"Idiopathic Hypersomnia","enrollment":214},{"nctId":"NCT05223166","phase":"PHASE3","title":"A Clinical Study to Evaluate the Efficacy and Safety of Pitolisant in the Treatment of EDS in Patients With OSA","status":"COMPLETED","sponsor":"Citrine Medicine Limited","startDate":"2022-04-12","conditions":"Excessive Daytime Sleepiness","enrollment":240},{"nctId":"NCT04026750","phase":"PHASE1","title":"Insulin Tolerance Test Study in Patients With Type 1 Diabetes","status":"TERMINATED","sponsor":"High Point Clinical Trials Center","startDate":"2019-09-15","conditions":"Type 1 Diabetes, Hypoglycemia","enrollment":5},{"nctId":"NCT00690274","phase":"PHASE2","title":"Study to Demonstrate Cognitive Enhancing Effects of BF2.649","status":"COMPLETED","sponsor":"University of Texas Southwestern Medical Center","startDate":"2008-06","conditions":"Schizophrenia","enrollment":52},{"nctId":"NCT03152123","phase":"PHASE1","title":"Determination the Abuse Potential of Pitolisant in Healthy, Non-Dependent Recreational Stimulant Users","status":"COMPLETED","sponsor":"Bioprojet","startDate":"2017-03-15","conditions":"Healthy, Drug Abuse","enrollment":43},{"nctId":"NCT01638403","phase":"PHASE3","title":"Effects of BF2.649 in the Treatment of Excessive Daytime Sleepiness in Narcolepsy.","status":"COMPLETED","sponsor":"Bioprojet","startDate":"2010-11","conditions":"Treatment of Excessive Daytime Sleepiness in Narcolepsy","enrollment":180},{"nctId":"NCT02800083","phase":"PHASE2","title":"A Trial Evaluating Pitolisant (BF2.649) in Alcohol Use Disorder Treatment","status":"WITHDRAWN","sponsor":"Bioprojet","startDate":"2016-10","conditions":"Alcohol Abuse, Nervous System","enrollment":""},{"nctId":"NCT01800045","phase":"PHASE3","title":"Pitolisant to Assess Weekly Frequency of Cataplexy Attacks and EDS in Narcoleptic Patients (HARMONY CTP)","status":"COMPLETED","sponsor":"Bioprojet","startDate":"2013-04","conditions":"Narcolepsy With Cataplexy, Excessive Daytime Sleepiness","enrollment":103},{"nctId":"NCT01789398","phase":"PHASE3","title":"Patient Narcoleptic Treated With BF2.649 (Pitolisant) in add-on to Sodium Oxybate (HARMONY IV)","status":"COMPLETED","sponsor":"Bioprojet","startDate":"2012-09","conditions":"Narcolepsy","enrollment":51},{"nctId":"NCT01072968","phase":"PHASE3","title":"BF2.649 in Patients With OSA, Still Complaining of EDS and Refusing to be Treated by CPAP.","status":"COMPLETED","sponsor":"Bioprojet","startDate":"2011-10","conditions":"Obstructive Sleep Apnea, Excessive Daytime Sleepiness","enrollment":268},{"nctId":"NCT01066442","phase":"PHASE3","title":"Efficacy and Safety of BF2.649 in Excessive Daytime Sleepiness (EDS) in Parkinson's Disease","status":"COMPLETED","sponsor":"Bioprojet","startDate":"2010-03","conditions":"Parkinson's Disease","enrollment":273},{"nctId":"NCT01036139","phase":"PHASE3","title":"Efficacy and Safety of BF2.649 in Excessive Daytime Sleepiness (EDS) in Parkinson's Disease","status":"COMPLETED","sponsor":"Bioprojet","startDate":"2009-12","conditions":"Parkinson's Disease","enrollment":268},{"nctId":"NCT01067235","phase":"PHASE3","title":"Efficacy and Safety Study of BF2.649 and BF2.649 Add on Modafinil on Cataplexy in Patients With Narcolespy","status":"COMPLETED","sponsor":"Bioprojet","startDate":"2009-10","conditions":"Narcolepsy, Cataplexy, Excessive Daytime Sleepiness","enrollment":14},{"nctId":"NCT01620554","phase":"PHASE2","title":"Dose-range Finding Study of BF2.649 Effect on Patients With Obstructive Sleep Apnea (OSA)","status":"COMPLETED","sponsor":"Bioprojet","startDate":"2010-10","conditions":"Obstructive Sleep Apnoea, Excessive Daytime Sleepiness","enrollment":110},{"nctId":"NCT01067222","phase":"PHASE3","title":"Efficacy and Safety Study of BF2.649 in the Treatment of Excessive Daytime Sleepiness in Narcolepsy","status":"COMPLETED","sponsor":"Bioprojet","startDate":"2009-05","conditions":"Narcolepsy, Excessive Daytime Sleepiness, Cataplexy","enrollment":110}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":0,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":[],"phase":"phase_3","status":"active","brandName":"Double-blind pitolisant","genericName":"Double-blind pitolisant","companyName":"Harmony Biosciences Management, Inc.","companyId":"harmony-biosciences-management-inc","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Pitolisant is a selective histamine H3 receptor antagonist that increases wakefulness by blocking inhibitory histamine signaling in the brain. Used for Narcolepsy type 1 and type 2, Idiopathic hypersomnia.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}