Idiopathic Hypersomnia
Conditions
Keywords
pitolisant, HBS-301, idiopathic hypersomnia, excessive daytime sleepiness, sleep inertia, fatigue
Brief summary
This is a Phase 3, multicenter, randomized, double-blind, parallel-group, placebo-controlled clinical study to assess the efficacy and safety of HBS-301 in adult participants (ages ≥18 years) with idiopathic hypersomnia (IH).
Detailed description
Approximately 248 participants are planned for randomization in the study. The study will consist of a Screening/Baseline Period (up to 28 days), a Double-blind Treatment Period (8 weeks), an optional Open-label Extension (OLE) Period (1 year), and 30 days of safety follow-up.
Interventions
DRUGHBS-301 tablet
HBS-301 tablet
DRUGPlacebo
Placebo tablet
Sponsors
Harmony Biosciences Management, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Has a current documented diagnosis of IH per the International Classification of Sleep Disorders, Third Edition (ICSD-3) or Text Revision (ICSD-3-TR) criteria with confirmatory polysomnogram (PSG) with multiple sleep latency test (MSLT; and if applicable, a 24-hour PSG report or an actigraphy report with sleep log) on file that led to the diagnosis and was completed within the last 10 years. * Has EDS. * Has moderate to very severe symptoms of IH. * If taking a permitted chronic concomitant medication or supplement, including nonprohibited antidepressants or wake-promoting agents, must be on a stable dose for at least 3 months prior to Screening and agree to continue at that stable dose for the Double-blind Treatment Period of the study. As-needed use of any treatment that could affect daytime sleepiness (including but not limited to stimulants, modafinil, and armodafinil) used on an as-needed basis is not permitted.
Exclusion criteria
* Has hypersomnia due to another medical disorder. * Has a history of pitolisant use within 5 half-lives prior to Screening. * Has a primary diagnosis of psychiatric illness, including depression, that is not well controlled. * Has a history of moderate or severe hepatic impairment. * Has a body surface area (BSA)-corrected estimated glomerular filtration rate (eGFR) \<60 mL/min. * Has a known history of long QT syndrome or any significant history of a serious abnormality of the electrocardiogram (ECG).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in severity of EDS as measured by the Epworth Sleepiness Scale (ESS) | Baseline to the end of the Double-blind Treatment Period (8 weeks) | The ESS is an 8-item, 4-point rating scale. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in severity of IH symptoms as measured by the Idiopathic Hypersomnia Severity Scale (IHSS) | Baseline to the end of the Double-blind Treatment Period (8 weeks) | The IHSS is a 14-item questionnaire designed to measure IH symptoms. |
| Change in sleep inertia as measured by the Sleep Inertia Questionnaire (SIQ) | Baseline to the end of the Double-blind Treatment Period (8 weeks) | The SIQ is a 22-item questionnaire that measures aspects of night and day sleep symptoms and the sleep inertia related to each. |
| Change in fatigue as measured by the Patient-Reported Outcomes Measurement Information System Fatigue Short Form 7a | Baseline to the end of the Double-blind Treatment Period (8 weeks) | The PROMIS-Fatigue-SF-7a is a 7-question, 5-point scale used to assess fatigue. |
| Change in severity of EDS as measured by the Epworth Sleepiness Scale | Baseline through Week 1 and Week 2 of the Titration Period (1 week and 2 weeks) | The ESS is an 8-item, 4-point rating scale. |
| Change in severity of IH symptoms as measured by the IHSS | Baseline through Week 1 and Week 2 of the Titration Period (1 week and 2 weeks) | The ESS is an 8-item, 4-point rating scale. |
| Change in severity of EDS as measured by the Clinical Global Impression of Severity (EDS) | Baseline to end of Double-blind Treatment Period (8 weeks) | The Clinical Global Impression of Severity (EDS) is a 5-item observer-rated scale that gauges the severity of a participant's EDS symptoms. |
| Change in severity of EDS as measured by the Patient Global Impression of Severity (EDS) | Baseline to the end of the Double-blind Treatment Period (8 weeks) | The Patient Global Impression of Severity (EDS) is a participant-reported assessment that gauges the severity of a participant's EDS symptoms. |
| Improvement in severity of EDS as measured by the Patient Global Impression of Change (EDS) | End of the Double-blind Treatment Period (8 weeks) | The Patient Global Impression of Change (EDS) is a participant-reported outcome used to evaluate the effectiveness of a treatment on their EDS symptoms. |
| Change in severity of IH symptoms as measured by the Clinical Global Impression of Severity (IH) | Baseline to end of Double-blind Treatment Period (8 weeks) | The Clinical Global Impression of Severity (IH) is a 3-item observer-rated scale used to track IH symptom changes. |
| Change in severity of IH symptoms as measured by the Patient Global Impression of Severity (IH) | Baseline to end of Double-blind Treatment Period (8 weeks) | The Patient Global Impression of Severity (IH) is a participant-reported assessment that gauges the severity of a participant's IH symptoms. |
| Improvement in severity of IH symptoms as measured by the Patient Global Impression of Change (IH) | Baseline to end of Double-blind Treatment Period (8 weeks) | The Patient Global Impression of Change (IH) is a participant-reported outcome used to evaluate the effectiveness of a treatment on their IH symptoms. |
| Change in severity of sleep inertia as measured by the Patient Global Impression of Severity (Sleep Inertia) | Baseline to end of Double-blind Treatment Period (8 weeks) | The Patient Global Impression of Severity (Sleep Inertia) is a participant-reported assessment that gauges the severity of a participant's sleep inertia symptoms. |
| Improvement in severity of sleep inertia as measured by the Patient Global Impression of Change (Sleep Inertia) | End of the Double-blind Treatment Period (8 weeks) | The Patient Global Impression of Change (Sleep Inertia) is a participant-reported outcome used to evaluate the effectiveness of a treatment on their sleep inertia symptoms. |
| Change in severity of fatigue as measured by the Patient Global Impression of Severity (Fatigue) | Baseline of the end of the Double-blind Treatment Period (8 weeks) | The Patient Global Impression of Severity (Fatigue) is a participant-reported assessment that gauges the severity of a participant's fatigue symptoms. |
| Improvement in severity of fatigue as measured by the Patient Global Impression of Change (Fatigue) | End of the Double-blind Treatment Period (8 weeks) | The Patient Global Impression of Change (Fatigue) is a participant-reported outcome used to evaluate the effectiveness of a treatment on their fatigue symptoms. |
| Change in cognitive complaints as measured by the British Columbia Cognitive Complaints Inventory | Baseline to the end of the Double-blind Treatment Period (8 weeks) | The British Columbia Cognitive Complaints Inventory is a participant-reported, 6-item, 4-point scale that assesses perceived cognitive difficulties. |
| Change in health-related quality of life as measured by the Short Form Health Survey-36 physical and mental component summaries | Baseline to the end of the Double-blind Treatment Period (8 weeks) | The Short Form-36 includes 36 questions across 8 health domains to measure a participant's functional health and well-being. |
| Change in work productivity as measured by the Work Productivity and Activity Impairment: Idiopathic Hypersomnia Work Productivity loss score | Baseline to the end of the Double-blind Treatment Period (8 weeks) | The Work Productivity and Activity Impairment: Idiopathic Hypersomnia questionnaire is a 6-item scale used to measure impairments over 7 days. |
| Incidence of treatment-emergent adverse events | Throughout study (16 months including OLE) | A treatment-emergent adverse event is any adverse event reported after the first dose of study drug and up to 30 days after final dose of study drug, or any worsening of a pre-existing condition reported after first dose of study drug and up to 30 days after final dose of study drug. |
Countries
Canada, United States
Contacts
CONTACTKatie Wilmsen
CONTACTMichelle Manuel
STUDY_DIRECTORDavid Seiden, MD
Harmony Biosciences Management, Inc.
Outcome results
None listed