A Long-Term Safety and Effectiveness Study to Evaluate Pitolisant in Adult Patients With Idiopathic Hypersomnia
- Conditions
- Idiopathic HypersomniaExcessive Daytime Sleepiness
- Interventions
- Registration Number
- NCT05458128
- Lead Sponsor
- Harmony Biosciences, LLC
- 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 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 & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 128
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Pitolisant 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.
- Primary Outcome Measures
Name Time Method Excessive daytime sleepiness Up to approximately 3 years 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.Safety and tolerability of pitolisant Up to approximately 3 years Incidence of adverse events (AEs)
- Secondary Outcome Measures
Name Time Method Functional outcomes of sleep Up to approximately 3 years 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.Sleep related impairments during wakefulness Up to approximately 3 years 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.Symptoms of idiopathic hypersomnia Up to approximately 3 years 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.Sleep inertia Up to approximately 3 years 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.
Trial Locations
- Locations (32)
Clinical Neurophysiology Services
🇺🇸Sterling Heights, Michigan, United States
Western Michigan University Homer Stryker MD School of Medicine
🇺🇸Kalamazoo, Michigan, United States
Sleep Dynamics
🇺🇸Neptune City, New Jersey, United States
Cedars-Sinai Medical Towers
🇺🇸Los Angeles, California, United States
Sleep Medicine Specialists of California
🇺🇸San Ramon, California, United States
Meris Clinical Research
🇺🇸Brandon, Florida, United States
St. Francis Medical Institute
🇺🇸Clearwater, Florida, United States
Florida Pediatric Research Institute
🇺🇸Winter Park, Florida, United States
Neurotrials Research Inc.
🇺🇸Atlanta, Georgia, United States
NorthShore University Health System
🇺🇸Glenview, Illinois, United States
OSF HealthCare Saint Francis Medical Center
🇺🇸Peoria, Illinois, United States
St. Luke's Sleep Medicine and Research Center
🇺🇸Chesterfield, Missouri, United States
Clayton Sleep Institute
🇺🇸Saint Louis, Missouri, United States
Great Plains Health
🇺🇸North Platte, Nebraska, United States
Northwell Health
🇺🇸New Hyde Park, New York, United States
Duke University School of Medicine
🇺🇸Durham, North Carolina, United States
Clinical Research of Gastonia
🇺🇸Gastonia, North Carolina, United States
ARSM Research
🇺🇸Huntersville, North Carolina, United States
NeuroScience Research Center, LLC
🇺🇸Canton, Ohio, United States
Intrepid Research, LLC
🇺🇸Cincinnati, Ohio, United States
Ohio Sleep Medicine and Neuroscience Institue
🇺🇸Dublin, Ohio, United States
North Star Medical Research
🇺🇸Middleburg, Ohio, United States
Abington Neurological Associates
🇺🇸Willow Grove, Pennsylvania, United States
Respiratory Specialists
🇺🇸Wyomissing, Pennsylvania, United States
Bogan Sleep Consultants
🇺🇸Columbia, South Carolina, United States
Lowcountry Lung Critical Care
🇺🇸North Charleston, South Carolina, United States
Neurology Clinic, P.C.
🇺🇸Cordova, Tennessee, United States
FutureSearch Trials of Neurology LP
🇺🇸Austin, Texas, United States
Central Texas Neurology Consultants, PA
🇺🇸Round Rock, Texas, United States
Comprehensive Sleep Medicine Associates
🇺🇸Sugar Land, Texas, United States
Northwest Houston Neurology and Sleep
🇺🇸Tomball, Texas, United States
University of Wisconsin-Madison
🇺🇸Madison, Wisconsin, United States