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

NCT ID: NCT05458128

Last Updated: 2025-12-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

119 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-08-19

Study Completion Date

2025-10-01

Brief Summary

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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.

Detailed Description

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This is a Phase 3 open-label study to evaluate the long-term safety and effectiveness of pitolisant in adult patients with IH. Patients who complete the Double-Blind Randomized Withdrawal Phase of study HBS-101-CL-010 (i.e., completed the End-of-Treatment \[EOT\] Visit/Visit 5 in the HBS-101-CL-010 study) and who continue to meet eligibility criteria for study HBS-101-CL-011 are eligible for enrollment.

Enrolled patients will be dispensed open-label pitolisant and may be titrated up to a maximum dose of 35.6 mg, based on the Investigator's assessment of safety/tolerability and effectiveness, during a 3-week Titration Period (Day 1 to Day 21) in accordance with the schedule:

* Week 1 (Day 1-7), 8.9 mg
* Week 2 (Day 8-14), 17.8 mg
* Week 3 (Day 15-21), 35.6 mg

The Long-Term Dosing Period will begin on Day 22 and will continue until the patient discontinues from the study or the Sponsor elects to terminate the study (i.e., End-of-Study \[EOS\]).

An on-site study visit will occur approximately 180 days after Visit 2 on Day 202 (Visit 3). On-site study visits will occur approximately every 6 months and telephone contacts (TCs) approximately every one month in between until the patient withdraws from the study or the study is terminated by the Sponsor. The dose of pitolisant may be adjusted (higher or lower) in increments of 4.45 mg starting at 8.9 mg up to 35.6 mg during the Long-Term Dosing Period based on Investigator assessment of safety/tolerability and effectiveness.

All patients will receive safety follow-up TCs from the study site 15 days and 30 days after their final dose of pitolisant, to assess for adverse events (AEs) and concomitant medication use.

Conditions

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Idiopathic Hypersomnia Excessive Daytime Sleepiness

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Pitolisant

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.

Group Type OTHER

Pitolisant

Intervention Type DRUG

Pitolisant 4.45 mg tablets

Pitolisant 17.8 mg tablets

Interventions

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Pitolisant

Pitolisant 4.45 mg tablets

Pitolisant 17.8 mg tablets

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Is able to provide voluntary, informed consent.
2. Completed the Double-Blind Randomized Withdrawal Phase (EOT/Visit 5) from the HBS-101-CL-010 study.
3. 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.
4. 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.
5. In the opinion of the Investigator, the patient is capable of understanding and complying with the protocol and administration of oral study drug.

Exclusion Criteria

1. Does not agree to discontinue any prohibited medication or substances listed in the protocol.
2. 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.
3. Participation in an interventional research study with an investigational medication or device, other than pitolisant, for the duration of the study.
4. 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).
5. Is receiving or is unable to discontinue a medication known to prolong the QT interval.
6. 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.
7. 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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Harmony Biosciences Management, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Cedars-Sinai Medical Towers

Los Angeles, California, United States

Site Status

Sleep Medicine Specialists of California

San Ramon, California, United States

Site Status

Meris Clinical Research

Brandon, Florida, United States

Site Status

St. Francis Medical Institute

Clearwater, Florida, United States

Site Status

Florida Pediatric Research Institute

Winter Park, Florida, United States

Site Status

Neurotrials Research Inc.

Atlanta, Georgia, United States

Site Status

NorthShore University Health System

Glenview, Illinois, United States

Site Status

OSF HealthCare Saint Francis Medical Center

Peoria, Illinois, United States

Site Status

Western Michigan University Homer Stryker MD School of Medicine

Kalamazoo, Michigan, United States

Site Status

Clinical Neurophysiology Services

Sterling Heights, Michigan, United States

Site Status

St. Luke's Sleep Medicine and Research Center

Chesterfield, Missouri, United States

Site Status

Clayton Sleep Institute

St Louis, Missouri, United States

Site Status

Great Plains Health

North Platte, Nebraska, United States

Site Status

Sleep Dynamics

Neptune City, New Jersey, United States

Site Status

Northwell Health

New Hyde Park, New York, United States

Site Status

Duke University School of Medicine

Durham, North Carolina, United States

Site Status

Clinical Research of Gastonia

Gastonia, North Carolina, United States

Site Status

ARSM Research

Huntersville, North Carolina, United States

Site Status

NeuroScience Research Center, LLC

Canton, Ohio, United States

Site Status

Intrepid Research, LLC

Cincinnati, Ohio, United States

Site Status

Ohio Sleep Medicine and Neuroscience Institue

Dublin, Ohio, United States

Site Status

North Star Medical Research

Middleburg, Ohio, United States

Site Status

Abington Neurological Associates

Willow Grove, Pennsylvania, United States

Site Status

Respiratory Specialists

Wyomissing, Pennsylvania, United States

Site Status

Bogan Sleep Consultants

Columbia, South Carolina, United States

Site Status

Lowcountry Lung Critical Care

North Charleston, South Carolina, United States

Site Status

Neurology Clinic, P.C.

Cordova, Tennessee, United States

Site Status

FutureSearch Trials of Neurology LP

Austin, Texas, United States

Site Status

Central Texas Neurology Consultants, PA

Round Rock, Texas, United States

Site Status

Comprehensive Sleep Medicine Associates

Sugar Land, Texas, United States

Site Status

Northwest Houston Neurology and Sleep

Tomball, Texas, United States

Site Status

University of Wisconsin-Madison

Madison, Wisconsin, United States

Site Status

Countries

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United States

Other Identifiers

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HBS-101-CL-011

Identifier Type: -

Identifier Source: org_study_id

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