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

Primary Purpose

Idiopathic Hypersomnia, Excessive Daytime Sleepiness

Status

Active

Phase

Phase 3

Locations

United States

Study Type

Interventional

Intervention

pitolisant

About this trial

This is an interventional treatment trial for Idiopathic Hypersomnia focused on measuring pitolisant, histamine, sleepiness, IH

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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.

Sites / Locations

Phoenix Medical Group
Mayo Clinic Arizona
Cedars-Sinai Medical Towers
University of California- Los Angeles
Stanford Center for Sleep Sciences and Medicine
Sleep Medicine Specialists of California
SDS Clinical Trials Inc.
Santa Monica Clinical Trials
Alpine Clinical Research Center
Norwalk Hospital Sleep Center
Meris Clinical Research
St. Francis Medical Institute
Sleep Medicine Specialists of South Florida, PA
Florida Pediatric Research Institute
Neurotrials Research Inc.
The Neurological Center of North GA
Northwestern University Feinberg School of Medicine
NorthShore University Health System
OSF HealthCare Saint Francis Medical Center
Helene A. Emsellem MD PC
Boston Children's Hospital
Neurocare, INC
Henry Ford Health System
Bronson Sleep Health
Clinical Neurophysiology Services
Minnesota Lung Center
Minnesota Lung Center
St. Luke's Sleep Medicine and Research Center
Clayton Sleep Institute
Great Plains Health
Neurology Specialists of Monmouth County, PA
Northwell Health
Research Carolina Elite LLC
Duke University School of Medicine
Clinical Research of Gastonia
ARSM Research
Raleigh Neurology Associates
Intrepid Research, LLC
Rainbow Babies Children's Hospital
Cleveland Clinc
Ohio Sleep Medicine and Neuroscience Institue
North Star Medical Research
CardioVoyage
Geisinger Medical Center
Brian Abaluck, LLC
Abington Neurological Associates
Respiratory Specialists
Medical University of South Carolina- Institute of Psychiatry
Bogan Sleep Consultants
Lowcountry Lung Critical Care
Advanced Center for Sleep Disorders
Neurology Clinic, P.C.
FutureSearch Trials of Neurology LP
Central Texas Neurology Consultants, PA
Comprehensive Sleep Medicine Associates
Northwest Houston Neurology and Sleep
Children's Hospital of the King's Daughter
University of Wisconsin-Madison

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

pitolisant

Arm Description

Open-label: 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.

Outcomes

Primary Outcome Measures

Safety and Tolerability of Pitolisant

The long term safety and tolerability of pitolisant will be evaluated via the incidence of adverse events (AEs) and changes in clinical laboratory test results, vital signs, 12-lead electrocardiograms (ECGs), and Columbia-Suicide Severity Rating Scale (C-SSRS) assessments.

Excessive Daytime Sleepiness

Change 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.

Symptoms of idiopathic hypersomnia

Change in Idiopathic Hypersomnia Severity Scale (IHSS). The score of the IHSS ranges from 0 to 50. A decrease in score represents an improvement in symptoms of idiopathic hypersomnia.

Symptoms of idiopathic hypersomnia

Change in Clinical Global Impression of Severity (CGI-S). The CGI-S is is a five item scale that ranges from none to very severe. An assessment of less severe symptoms represents an improvement in the clinician's perception of the patient's overall clinical status related to idiopathic hypersomnia.

Symptoms of idiopathic hypersomnia

Change in Patient Global Impression of Severity (PGI-S) of their excessive daytime sleepiness. The PGI-S is a five item scale that ranges from none to very severe. An assessment of less severe symptoms represents an improvement in the patient's perception of the severity of their excessive daytime sleepiness.

Functional outcomes of sleep

Change in Functional Outcomes of Sleep Questionnaire 10-item version (FOSQ-10). The score of the FOSQ-10 ranges from 5 to 20. An increase in score represents an improvement in the patient's impression of the impact of hypersomnia on multiple activities of everyday living.

Sleep related impairments during wakefulness

Change in Patient-Reported Outcomes Measurement Information System, Sleep-Related Impairment (PROMIS-SRI). The score of the PROMIS-SRI ranges from 8-40. A decrease in score represents an improvement in the patient's impression of the impact of hypersomnia on multiple activities of everyday living.

Sleep inertia

Change in Sleep Inertia Questionnaire (SIQ). The SIQ ranges from 21 to 105. A decrease in score represents an improvement in the patient's ability to wake up after sleep.

Secondary Outcome Measures

Full Information

NCT ID

NCT05458128

First Posted

July 11, 2022

Last Updated

August 30, 2023

Sponsor

Harmony Biosciences, LLC

1. Study Identification

Unique Protocol Identification Number

NCT05458128

Brief Title

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

Official Title

An Open-Label Study to Evaluate the Long-Term Safety and Effectiveness of Pitolisant in Adult Patients With Idiopathic Hypersomnia Who Completed Study HBS-101-CL-010

Study Type

Interventional

2. Study Status

Record Verification Date

August 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

August 19, 2022 (Actual)

Primary Completion Date

August 2025 (Anticipated)

Study Completion Date

December 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Harmony Biosciences, LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

Detailed Description

This is a Phase 3 open-label, long-term 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 meet the inclusion/exclusion 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 (±3 days) 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 (±7 days; 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 (±3) days and 30 (+3) days after their final dose of pitolisant, to assess for adverse events (AEs) and concomitant medication use.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Idiopathic Hypersomnia, Excessive Daytime Sleepiness

Keywords

pitolisant, histamine, sleepiness, IH

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

128 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

pitolisant

Arm Type

Other

Arm Description

Intervention Type

Drug

Intervention Name(s)

pitolisant

Intervention Description

Pitolisant 4.45 mg tablets: white, round, plain, biconvex film-coated tablet, 3.7 mm in diameter. The 4.45 mg strength tablet may be marked with "S" on one side and is plain on the other side. Each tablet contains 5 mg of pitolisant hydrochloride equivalent to 4.45 mg of pitolisant. Pitolisant 17.8 mg: white, round, plain, biconvex film-coated tablet, 7.5 mm in diameter. The 17.8 mg strength tablet may be marked with "H" on one side and is plain on the other side. Each tablet contains 20 mg of pitolisant hydrochloride equivalent to 17.8 mg of pitolisant.

Primary Outcome Measure Information:

Title

Safety and Tolerability of Pitolisant

Description

Time Frame

Screening to 30 (+3) days after final dose

Title

Excessive Daytime Sleepiness

Description

Change 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.

Time Frame