A Long-Term Safety and Effectiveness Study to Evaluate Pitolisant in Adult Patients With Idiopathic Hypersomnia

NCT05458128 | Completed Phase 3 FDA Drug

• 1 other identifier: HBS-101-CL-011
• Study Type: interventional
• Target: 119
• Locations: 1 country
• Sites: 32

Brief Summary

The primary objective of this study is to assess the long-term safety and effectiveness of pitolisant in patients with idiopathic hypersomnia (IH) who completed the Double-Blind Randomized Withdrawal Phase of study HBS-101-CL-010.

Conditions

Excessive Daytime Sleepiness; Idiopathic Hypersomnia

Keywords

pitolisant; histamine; sleepiness; IH

Outcome Measures

Primary Outcomes (2)

• Safety and tolerability of pitolisant

  Incidence of adverse events (AEs)

  Up to approximately 3 years

• Excessive daytime sleepiness

  Change from Baseline in Epworth Sleepiness Scale (ESS) score The score of the Epworth Sleepiness Scale ranges from 0 to 24. A decrease in score represents an improvement in excessive daytime sleepiness.

  Up to approximately 3 years

Secondary Outcomes (6)

• Symptoms of idiopathic hypersomnia

  Up to approximately 3 years

• Symptoms of idiopathic hypersomnia

  Up to approximately 3 years

• Symptoms of idiopathic hypersomnia

  Up to approximately 3 years

• Functional outcomes of sleep

  Up to approximately 3 years

• Sleep related impairments during wakefulness

  Up to approximately 3 years

Study Arms (1)

Pitolisant

OTHER

Week 1: 8.9 mg pitolisant administered once daily in the morning upon wakening; Week 2: 17.8 mg pitolisant administered once daily in the morning upon wakening; Weeks 3 through end of treatment: 17.8 mg to 35.6 mg pitolisant administered once daily in the morning upon wakening.

Drug: Pitolisant

Interventions

Pitolisant DRUG

Pitolisant 4.45 mg tablets Pitolisant 17.8 mg tablets

Pitolisant

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Is able to provide voluntary, informed consent.
• Completed the Double-Blind Randomized Withdrawal Phase (EOT/Visit 5) from the HBS-101-CL-010 study.
• A patient who is a female of child-bearing potential must have a negative urine pregnancy test at the Screening Visit. A patient who is a female of child-bearing potential must agree to remain abstinent or use an effective method of non-hormonal contraception to prevent pregnancy for the duration of the study and for 21 days after final dose of study drug.
• Must have a negative result on urine drug screen at the Screening Visit, except for medications that are prescribed by a healthcare provider for medical conditions.
• In the opinion of the Investigator, the patient is capable of understanding and complying with the protocol and administration of oral study drug.

You may not qualify if:

• Does not agree to discontinue any prohibited medication or substances listed in the protocol.
• Is currently breastfeeding or planning to breastfeed over the course of the study. Lactating women must agree not to breastfeed for the duration of the study and for 21 days after final dose of study drug.
• Participation in an interventional research study with an investigational medication or device, other than pitolisant, for the duration of the study.
• Has a diagnosis of end-stage renal disease (ESRD; estimated glomerular filtration rate \[eGFR\] of \<15 mL/minute/1.73 m2) or severe hepatic impairment (Child-Pugh C).
• Is receiving or is unable to discontinue a medication known to prolong the QT interval.
• Has a significant risk of committing suicide or suicidality based on history; routine psychiatric examination; Investigator's judgment; or who has an answer of "yes" on any question other than questions 1 to 3 or "yes" on any question in the suicidal behavior section of the C-SSRS, Since Last Visit.
• Based on the judgment of the Investigator, is unsuitable for the study for any reason, including but not limited to an unstable or uncontrolled medical condition or one that might interfere with the conduct of the study, confound interpretation of study results, pose a health risk to the patient, or compromise the integrity of the study.

Sponsors & Collaborators

• Harmony Biosciences Management, Inc.: lead

Study Sites (32)

Cedars-Sinai Medical Towers

Los Angeles, California, 90048, United States

Location

Sleep Medicine Specialists of California

San Ramon, California, 94583, United States

Location

Meris Clinical Research

Brandon, Florida, 33511, United States

Location

St. Francis Medical Institute

Clearwater, Florida, 33765, United States

Location

Florida Pediatric Research Institute

Winter Park, Florida, 32789, United States

Location

Neurotrials Research Inc.

Atlanta, Georgia, 30328, United States

Location

NorthShore University Health System

Glenview, Illinois, 60026, United States

Location

OSF HealthCare Saint Francis Medical Center

Peoria, Illinois, 61637, United States

Location

Western Michigan University Homer Stryker MD School of Medicine

Kalamazoo, Michigan, 49008, United States

Location

Clinical Neurophysiology Services

Sterling Heights, Michigan, 48314, United States

Location

St. Luke's Sleep Medicine and Research Center

Chesterfield, Missouri, 63017, United States

Location

Clayton Sleep Institute

St Louis, Missouri, 63123, United States

Location

Great Plains Health

North Platte, Nebraska, 69101, United States

Location

Sleep Dynamics

Neptune City, New Jersey, 07753, United States

Location

Northwell Health

New Hyde Park, New York, 11042, United States

Location

Duke University School of Medicine

Durham, North Carolina, 27705, United States

Location

Clinical Research of Gastonia

Gastonia, North Carolina, 28054, United States

Location

ARSM Research

Huntersville, North Carolina, 28078, United States

Location

NeuroScience Research Center, LLC

Canton, Ohio, 44718, United States

Location

Intrepid Research, LLC

Cincinnati, Ohio, 45245, United States

Location

Ohio Sleep Medicine and Neuroscience Institue

Dublin, Ohio, 43017, United States

Location

North Star Medical Research

Middleburg, Ohio, 44130, United States

Location

Abington Neurological Associates

Willow Grove, Pennsylvania, 19090, United States

Location

Respiratory Specialists

Wyomissing, Pennsylvania, 19610, United States

Location

Bogan Sleep Consultants

Columbia, South Carolina, 29201, United States

Location

Lowcountry Lung Critical Care

North Charleston, South Carolina, 29406, United States

Location

Neurology Clinic, P.C.

Cordova, Tennessee, 38018, United States

Location

FutureSearch Trials of Neurology LP

Austin, Texas, 78731, United States

Location

Central Texas Neurology Consultants, PA

Round Rock, Texas, 78681, United States

Location

Comprehensive Sleep Medicine Associates

Sugar Land, Texas, 77478, United States

Location

Northwest Houston Neurology and Sleep

Tomball, Texas, 77375, United States

Location

University of Wisconsin-Madison

Madison, Wisconsin, 53719, United States

Location

MeSH Terms

Conditions

Idiopathic Hypersomnia; Disorders of Excessive Somnolence; Sleepiness

Interventions

pitolisant

Condition Hierarchy (Ancestors)

Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Nervous System Diseases; Mental Disorders; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 11, 2022
• First Posted: July 14, 2022
• Study Start: August 19, 2022
• Primary Completion: October 1, 2025
• Study Completion: October 1, 2025
• Last Updated: December 12, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (32)