A Phase 3 Efficacy and Safety Study of HBS-301 in Participants With Idiopathic Hypersomnia (IH)

Phase 3RecruitingNCT07500090

Harmony Biosciences Management, Inc.248 enrolled

Overview

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 treating idiopathic hypersomnia (IH) symptoms, including excessive daytime sleepiness (EDS), sleep inertia, and fatigue in adult participants (ages ≥18 years) with idiopathic hypersomnia (IH). The primary objective of this study is to evaluate the efficacy of HBS-301 compared with placebo in treating IH symptoms. Secondary objectives include evaluating the efficacy of HBS-301 compared with placebo in treating EDS, sleep inertia, and fatigue.

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 treating IH symptoms, including EDS, sleep inertia, and fatigue in adult participants (ages ≥18 years) with IH. 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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

248

Conditions

Idiopathic Hypersomnia

Interventions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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, an actigraphy report with sleep log on file that led to the diagnosis and was completed within the last 10 years. * Has moderate to very severe symptoms of IH. * If taking a permitted chronic concomitant medication or supplement, including nonprohibited antidepressants, 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. 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. * For participants being treated for obstructive sleep apnea (OSA) or other hypoventilatory conditions, must be compliant with their medical device or oral appliance as determined by the Investigator. Participants must maintain OSA treatment compliance throughout the study. 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 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).

Locations (12)

Santa Monica Clinical Trials

Santa Monica, California, United States

RECRUITING

PharmDev Research Institute, LLC

Miami, Florida, United States

RECRUITING

Central Florida Pediatric Sleep Disorders Institute (Florida Pediatric Research Institute, LLC)

Winter Park, Florida, United States

RECRUITING

NeuroTrials Research Inc.

Atlanta, Georgia, United States

RECRUITING

Phillip Nowlin

Stockbridge, Georgia, United States

RECRUITING

St. Luke's Hospital, Sleep Medicine and Research Center

Chesterfield, Missouri, United States

RECRUITING

Clinical Research of Gastonia

Gastonia, North Carolina, United States

RECRUITING

Stern Research Partners, LLC

Huntersville, North Carolina, United States

RECRUITING

David Kudrow, MD

Morrisville, North Carolina, United States

RECRUITING

Respiratory Specialists

Wyomissing, Pennsylvania, United States

RECRUITING

...and 2 more locations

Outcomes

Primary Outcomes

To evaluate the efficacy of HBS-301 compared with placebo in treating IH symptoms

Change in severity of IH symptoms as measured by the Idiopathic Hypersomnia Severity Scale

Time frame: Baseline to the end of the Double-blind Treatment Period (8 weeks)

Secondary Outcomes

To evaluate the efficacy of HBS-301 compared with placebo in treating EDS

Change in severity of EDS as measured by the Epworth Sleepiness Scale

Time frame: Baseline to the end of the Double-blind Treatment Period (8 weeks)

To evaluate the efficacy of HBS-301 compared with placebo in treating sleep inertia

Change in sleep inertia as measured by the Sleep Inertia Questionnaire

Time frame: Baseline to the end of the Double-blind Treatment Period (8 weeks)

To evaluate the efficacy of HBS-301 compared with placebo on fatigue

Change in fatigue as measured by the Patient-Reported Outcomes Measurement Information System Fatigue Short Form 7a

Time frame: Baseline to the end of the Double-blind Treatment Period (8 weeks)

To evaluate the onset of efficacy of HBS-301 compared with placebo in treating EDS

Change in severity of EDS as measured by the Epworth Sleepiness Scale

Time frame: Baseline through Week 1 and Week 2 of the Titration Period (1 week and 2 weeks)

To evaluate the onset of efficacy of HBS-301 compared to placebo in treating IH symptoms

Change in severity of IH symptoms as measured by the Idiopathic Hypersomnia Severity Scale

Time frame: Baseline through Week 1 and Week 2 of the Titration Period (1 week and 2 weeks)

To evaluate the efficacy of HBS-301 compared with placebo in treating IH symptoms

Change in severity of IH symptoms as measured by the Clinical Global Impression of Severity (IH)

Time frame: Baseline to end of Double-blind Treatment Period (8 weeks)

To evaluate the efficacy of HBS-301 compared with placebo in treating IH symptoms

Change in severity of IH symptoms as measured by the Patient Global Impression of Severity (IH)

Time frame: Baseline to end of Double-blind Treatment Period (8 weeks)

To evaluate the efficacy of HBS-301 compared with placebo in treating sleep-related impairment

Change in severity of sleep-related impairment as measured by the PROMIS Custom Form v1.0 Sleep-Related Impairment-Harmony Biosciences

Time frame: Baseline to the end of the Double-blind Treatment Period (8 weeks)

To evaluate the efficacy of HBS-301 compared with placebo on severity of fatigue

Change in severity of fatigue as measured by the Patient Global Impression of Severity (Fatigue)

Time frame: Baseline of the end of the Double-blind Treatment Period (8 weeks)

To evaluate the efficacy of HBS-301 compared with placebo on overall severity of EDS

Change in severity of EDS as measured by the Patient Global Impression of Severity (EDS)

Time frame: Baseline to the end of the Double-blind Treatment Period (8 weeks)

To evaluate the efficacy of HBS-301 compared with placebo on severity of sleep inertia

Change in severity of sleep inertia as measured by the Patient Global Impression of Severity (Sleep Inertia)

Time frame: Baseline to end of Double-blind Treatment Period (8 weeks)

To evaluate the efficacy of HBS-301 compared with placebo on cognitive complaints

Change in cognitive complaints as measured by the British Columbia Cognitive Complaints Inventory

Time frame: Baseline to the end of the Double-blind Treatment Period (8 weeks)

To evaluate the efficacy of HBS-301 compared with placebo on overall health-related quality of life

Change in health-related quality of life as measured by the Short Form Health Survey-36

Time frame: Baseline to the end of the Double-blind Treatment Period (8 weeks)

To evaluate the efficacy of HBS-301 compared with placebo on work productivity

Change in work productivity as measured by the Work Productivity and Activity Impairment: Idiopathic Hypersomnia

Time frame: Baseline to the end of the Double-blind Treatment Period (8 weeks)

To evaluate the safety of HBS-301

Incidence of treatment-emergent adverse events

Time frame: Throughout study (16 months including OLE)

To evaluate the pharmacokinetic concentrations of pitolisant and major identified metabolites

Concentrations of pitolisant and major identified metabolites

Time frame: Throughout study (16 weeks)

A Phase 3 Efficacy and Safety Study of HBS-301 in Participants With Idiopathic Hypersomnia (IH)

Overview

Conditions

Interventions

Eligibility

Locations (12)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts