A Long-Term Safety and Effectiveness Study to Evaluate Pitolisant in Adult Patients With Idiopathic Hypersomnia
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| ClinicalTrials.gov Identifier: NCT05458128 |
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Recruitment Status :
Active, not recruiting
First Posted : July 14, 2022
Last Update Posted : January 25, 2024
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Idiopathic Hypersomnia Excessive Daytime Sleepiness | Drug: Pitolisant | Phase 3 |
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 Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 128 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| 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 |
| Actual Study Start Date : | August 19, 2022 |
| Estimated Primary Completion Date : | August 2025 |
| Estimated Study Completion Date : | December 2025 |
| Arm | Intervention/treatment |
|---|---|
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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.
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Drug: Pitolisant
Pitolisant 4.45 mg tablets Pitolisant 17.8 mg tablets |
- 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.
- Symptoms of idiopathic hypersomnia [ Time Frame: Up to approximately 3 years ]
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.
- Symptoms of idiopathic hypersomnia [ Time Frame: Up to approximately 3 years ]
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.
- Symptoms of idiopathic hypersomnia [ Time Frame: 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.
- Functional outcomes of sleep [ Time Frame: 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 [ Time Frame: 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.
- Sleep inertia [ Time Frame: 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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05458128
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| Responsible Party: | Harmony Biosciences, LLC |
| ClinicalTrials.gov Identifier: | NCT05458128 |
| Other Study ID Numbers: |
HBS-101-CL-011 |
| First Posted: | July 14, 2022 Key Record Dates |
| Last Update Posted: | January 25, 2024 |
| Last Verified: | January 2024 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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pitolisant histamine sleepiness IH |
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Disorders of Excessive Somnolence Idiopathic Hypersomnia Sleepiness Sleep Disorders, Intrinsic |
Dyssomnias Sleep Wake Disorders Nervous System Diseases Mental Disorders |