Idiopathic HypersomniaPhase 3NCT05458128
A Long-Term Safety and Effectiveness Study to Evaluate Pitolisant in Adult Patients With Idiopathic Hypersomnia
A Phase 3 study testing Pitolisant in people with Idiopathic Hypersomnia and Excessive Daytime Sleepiness, run by Harmony Biosciences Management, Inc. at 32 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Idiopathic Hypersomnia, Excessive Daytime Sleepiness
- What is being tested?
- Pitolisant
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 119 participants
- Where
- 32 sitesUnited States
- Study dates
- 19 Aug 2022to 1 Oct 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
32 sites in 1 country.
32 sites in 1 country
United States32 sites
Neurotrials Research Inc.
Atlanta, Georgia, 30328
FutureSearch Trials of Neurology LP
Austin, Texas, 78731
Meris Clinical Research
Brandon, Florida, 33511
NeuroScience Research Center, LLC
Canton, Ohio, 44718
St. Luke's Sleep Medicine and Research Center
Chesterfield, Missouri, 63017
Intrepid Research, LLC
Cincinnati, Ohio, 45245
St. Francis Medical Institute
Clearwater, Florida, 33765
Bogan Sleep Consultants
Columbia, South Carolina, 29201
Neurology Clinic, P.C.
Cordova, Tennessee, 38018
Ohio Sleep Medicine and Neuroscience Institue
Dublin, Ohio, 43017
Duke University School of Medicine
Durham, North Carolina, 27705
Clinical Research of Gastonia
Gastonia, North Carolina, 28054
NorthShore University Health System
Glenview, Illinois, 60026
ARSM Research
Huntersville, North Carolina, 28078
Western Michigan University Homer Stryker MD School of Medicine
Kalamazoo, Michigan, 49008
Cedars-Sinai Medical Towers
Los Angeles, California, 90048
University of Wisconsin-Madison
Madison, Wisconsin, 53719
North Star Medical Research
Middleburg, Ohio, 44130
Sleep Dynamics
Neptune City, New Jersey, 07753
Northwell Health
New Hyde Park, New York, 11042
Lowcountry Lung Critical Care
North Charleston, South Carolina, 29406
Great Plains Health
North Platte, Nebraska, 69101
OSF HealthCare Saint Francis Medical Center
Peoria, Illinois, 61637
Central Texas Neurology Consultants, PA
Round Rock, Texas, 78681
Sleep Medicine Specialists of California
San Ramon, California, 94583
Clayton Sleep Institute
St Louis, Missouri, 63123
Clinical Neurophysiology Services
Sterling Heights, Michigan, 48314
Comprehensive Sleep Medicine Associates
Sugar Land, Texas, 77478
Northwest Houston Neurology and Sleep
Tomball, Texas, 77375
Abington Neurological Associates
Willow Grove, Pennsylvania, 19090
Florida Pediatric Research Institute
Winter Park, Florida, 32789
Respiratory Specialists
Wyomissing, Pennsylvania, 19610
About this study
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
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.
Read the 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.
- Being tested
- Pitolisant
- Keywords
- pitolisant, histamine, sleepiness, IH
- Medical subject headings
- Idiopathic Hypersomnia, Disorders of Excessive Somnolence, Sleepiness
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PitolisantDrug
Pitolisant 4.45 mg tablets
Pitolisant 17.8 mg tablets
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Pitolisant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of pitolisant
Incidence of adverse events (AEs)
Measured: 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.
Measured: Up to approximately 3 years
Secondary outcomes (6)
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.
Measured: Up to approximately 3 years
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.
Measured: Up to approximately 3 years
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.
Measured: Up to approximately 3 years
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.
Measured: Up to approximately 3 years
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.
Measured: Up to approximately 3 years
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.
Measured: Up to approximately 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jul 2022
- Study start19 Aug 2022
- Primary completion1 Oct 2025
- Study completion1 Oct 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic HypersomniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 119 participants (actual)
Sponsor and investigators
- Lead sponsor
- Harmony Biosciences Management, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05458128
- Sponsor's protocol ID
- HBS-101-CL-011
- Status last verified
- Dec 2025
- First submitted
- 11 Jul 2022
- Last updated
- 12 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05458128
- Acronym / sponsor ID
- HBS-101-CL-011
- Keywords
- pitolisant, histamine, sleepiness, IH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 119 participants (actual)
- Lead sponsor
- Harmony Biosciences Management, Inc.Industry (pharma, biotech, devices)
- Study start
- 19 Aug 2022 (actual)
- Primary completion
- 1 Oct 2025 (actual)
- Study completion
- 1 Oct 2025 (actual)
- First posted
- 14 Jul 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pitolisant | Other | Drug: PitolisantWeek 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PitolisantPitolisant 4.45 mg tablets Pitolisant 17.8 mg tablets | Drug | Pitolisant |
Primary outcome
Safety and tolerability of pitolisant
Time frame: Up to approximately 3 years
Incidence of adverse events (AEs)
Excessive daytime sleepiness
Time frame: 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.
6 secondary outcomes
Symptoms of idiopathic hypersomnia
Time frame: Up to approximately 3 years
Symptoms of idiopathic hypersomnia
Time frame: Up to approximately 3 years
Symptoms of idiopathic hypersomnia
Time frame: Up to approximately 3 years
Functional outcomes of sleep
Time frame: Up to approximately 3 years
Sleep related impairments during wakefulness
Time frame: Up to approximately 3 years
Sleep inertia
Time frame: Up to approximately 3 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Dec 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
32 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Pitolisant in people with Idiopathic Hypersomnia and Excessive Daytime Sleepiness, run by Harmony Biosciences Management, Inc. at 32 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of pitolisant (time frame: Up to approximately 3 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Dec 2025.