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

Idiopathic Hypersomnia Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05458128
• Other study ID #: HBS-101-CL-011
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: August 19, 2022
• Est. completion date: December 2025

Study information

• Verified date: January 2024
• Source: Harmony Biosciences, LLC
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 128
• Est. completion date: December 2025
• Est. primary completion date: August 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Disorders of Excessive Somnolence
• Excessive Daytime Sleepiness
• Idiopathic Hypersomnia
• Sleepiness

Intervention

Drug:
• Pitolisant
Pitolisant 4.45 mg tablets Pitolisant 17.8 mg tablets

Locations

Country	Name	City	State
United States	Neurotrials Research Inc.	Atlanta	Georgia
United States	FutureSearch Trials of Neurology LP	Austin	Texas
United States	Alpine Clinical Research Center	Boulder	Colorado
United States	Meris Clinical Research	Brandon	Florida
United States	NeuroScience Research Center, LLC	Canton	Ohio
United States	Advanced Center for Sleep Disorders	Chattanooga	Tennessee
United States	St. Luke's Sleep Medicine and Research Center	Chesterfield	Missouri
United States	Intrepid Research, LLC	Cincinnati	Ohio
United States	St. Francis Medical Institute	Clearwater	Florida
United States	Bogan Sleep Consultants	Columbia	South Carolina
United States	Neurology Clinic, P.C.	Cordova	Tennessee
United States	Research Carolina Elite LLC	Denver	North Carolina
United States	Ohio Sleep Medicine and Neuroscience Institue	Dublin	Ohio
United States	Duke University School of Medicine	Durham	North Carolina
United States	Minnesota Lung Center	Edina	Minnesota
United States	Clinical Research of Gastonia	Gastonia	North Carolina
United States	NorthShore University Health System	Glenview	Illinois
United States	ARSM Research	Huntersville	North Carolina
United States	Cedars-Sinai Medical Towers	Los Angeles	California
United States	University of Wisconsin-Madison	Madison	Wisconsin
United States	Sleep Medicine Specialists of South Florida, PA	Miami	Florida
United States	North Star Medical Research	Middleburg	Ohio
United States	Northwell Health	New Hyde Park	New York
United States	Lowcountry Lung Critical Care	North Charleston	South Carolina
United States	Great Plains Health	North Platte	Nebraska
United States	Henry Ford Health System	Novi	Michigan
United States	OSF HealthCare Saint Francis Medical Center	Peoria	Illinois
United States	Bronson Sleep Health	Portage	Michigan
United States	Central Texas Neurology Consultants, PA	Round Rock	Texas
United States	Clayton Sleep Institute	Saint Louis	Missouri
United States	Sleep Medicine Specialists of California	San Ramon	California
United States	SDS Clinical Trials Inc.	Santa Ana	California
United States	Clinical Neurophysiology Services	Sterling Heights	Michigan
United States	Comprehensive Sleep Medicine Associates	Sugar Land	Texas
United States	Northwest Houston Neurology and Sleep	Tomball	Texas
United States	Neurology Specialists of Monmouth County, PA	West Long Branch	New Jersey
United States	Abington Neurological Associates	Willow Grove	Pennsylvania
United States	Florida Pediatric Research Institute	Winter Park	Florida
United States	Respiratory Specialists	Wyomissing	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Harmony Biosciences, LLC

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and tolerability of pitolisant	Incidence of adverse events (AEs)	Up to approximately 3 years
Primary	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	Symptoms of idiopathic hypersomnia	Change from Baseline 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.	Up to approximately 3 years
Secondary	Symptoms of idiopathic hypersomnia	Change from Baseline in Clinical Global Impression of Severity (CGI-S) for IH; The CGI-S 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.	Up to approximately 3 years
Secondary	Symptoms of idiopathic hypersomnia	Change from Baseline in Patient Global Impression of Severity (PGI-S) for EDS; 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.	Up to approximately 3 years
Secondary	Functional outcomes of sleep	Change from Baseline 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.	Up to approximately 3 years
Secondary	Sleep related impairments during wakefulness	Change from Baseline in Patient-Reported Outcomes Measurement Information System Sleep-Related Impairment Item Bank v1.0-Short Form 8a (PROMIS-SRI 8a); The score of the PROMIS-SRI 8a 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.	Up to approximately 3 years
Secondary	Sleep inertia	Change from Baseline 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.	Up to approximately 3 years