A Phase 3 Efficacy and Safety Study of HBS-301 in Participants With Narcolepsy

Phase 3RecruitingNCT07675135

Harmony Biosciences Management, Inc.258 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 excessive daytime sleepiness (EDS), cataplexy, sleepiness/wakefulness, and fatigue in adult participants (ages ≥18 years) with narcolepsy.

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 EDS, cataplexy, sleepiness/wakefulness, and fatigue in adult participants (ages ≥18 years) with narcolepsy. Approximately 258 participants are planned for randomization into 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 Period (1 year), and 30 days of safety follow-up.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

258

Conditions

EDS Cataplexy Fatigue Narcolepsy

Interventions

HBS-301DRUG

HBS-301 tablet

PlaceboOTHER

Placebo tablet

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Has a current documented diagnosis of NT1 or NT2 per the International Classification of Sleep Disorders, Third Edition (ICSD-3) or the ICSD-3 Text Revision (ICSD-3-TR) within the last 10 years. * Has EDS. * 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 (PRN) use of any treatment that could affect daytime sleepiness (including but not limited to oxybates, stimulants, modafinil, and armodafinil) 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 (i.e., symptoms and medications have not been stable for at least 3 months prior to Screening). * Has any history of bipolar disorder or psychosis * Has acute or chronic liver disease or a history of moderate or severe hepatic impairment. * Has a body surface area-corrected estimated glomerular filtration rate (eGFR) \<60 mL/min. * Has a known history of long QT syndrome or serious abnormality of the electrocardiogram (ECG).

Locations (53)

Outcomes

Primary Outcomes

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

The ESS is an 8-item, 4-point rating scale.

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

Secondary Outcomes

Change in Weekly Rate of Cataplexy (WRC) in participants with an average WRC of ≥3 over 2 consecutive weeks at Screening

The WRC is the average number of cataplexy attacks per week.

Time frame: End of the Double-Blind Treatment Period (8 weeks)

Change in sleepiness/wakefulness measured by the Maintenance of Wakefulness Test (MWT)

The MWT consists of a series of 20-minute to 40-minute trials spaced 2 hours apart and is used to measure an individual's ability to stay awake.

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

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

The PROMIS-Fatigue-SF-7a is a 7-question, 5-point scale used to assess fatigue.

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

Change in severity of EDS as measured by the ESS

The ESS is an 8-item, 4-point rating scale.

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

Onset of efficacy of HBS-301 compared with placebo in treating cataplexy measured by WRC in participants with an average WRC of ≥3 over 2 consecutive weeks during Screening

The WRC is the average number of cataplexy attacks per week.

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

Central Contacts

Michelle Manuel

CONTACT

847-903-4610 clinicaltrials@harmonybiosciences.com

Katie Wilmsen

CONTACT

443-309-5556 clinicaltrials@harmonybiosciences.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

Sleep Disorders Center of Alabama

Birmingham, Alabama, United States

NOT_YET_RECRUITING

Springhill Medical Center

Mobile, Alabama, United States

NOT_YET_RECRUITING

Phoenix Medical Group, PC

Peoria, Arizona, United States

RECRUITING

West Coast Pulmonary & Sleep Disorders Center

Lancaster, California, United States

NOT_YET_RECRUITING

Santa Monica Clinical Trials

Los Angeles, California, United States

RECRUITING

Stanford Center for Sleep Sciences and Medicine

Redwood City, California, United States

RECRUITING

University of California San Francisco

San Francisco, California, United States

NOT_YET_RECRUITING

Trial Site Solutions

San Ramon, California, United States

RECRUITING

Alpine Clinical Research Center, Inc

Boulder, Colorado, United States

RECRUITING

Delta Waves LLC

Colorado Springs, Colorado, United States

RECRUITING

...and 43 more locations

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

The Clinical Global Impression of Change (EDS) is a 7-point scale used to measure the improvement or worsening of the participant's EDS relative to a baseline.

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

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

The Patient Global Impression of Severity (EDS) is a participant-reported assessment that gauges the severity of a participant's EDS symptoms.

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

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

The Patient Global Impression of Change (EDS) is a participant-reported outcome used to evaluate the effectiveness of a treatment on their EDS symptoms.

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

Change in severity of cataplexy as measured by the Clinical Global Impression of Severity (Cataplexy) in participants with an average WRC of ≥3 over 2 consecutive weeks during Screening

The Clinical Global Impression of Severity (Cataplexy) is a 3-item observer-rated scale used to track cataplexy symptom changes.

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

Change in severity of cataplexy as measured by the Patient Global Impression of Severity (Cataplexy) in participants with an average WRC of ≥3 over 2 consecutive weeks during Screening

The Patient Global Impression of Severity (Cataplexy) is a participant-reported assessment that gauges the severity of cataplexy symptoms.

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

Improvement in severity of cataplexy as measured by the Patient Global Impression of Change (Cataplexy) in participants with an average WRC of ≥3 over 2 consecutive weeks during Screening

The Patient Global Impression of Change (Cataplexy) is a participant-reported outcome used to evaluate the effectiveness of a treatment on their cataplexy symptoms.

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

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

The Patient Global Impression of Severity (Fatigue) is a participant-reported assessment that gauges the severity of a participant's fatigue symptoms.

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

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

The Patient Global Impression of Change (Fatigue) is a participant-reported outcome used to evaluate the effectiveness of a treatment on their fatigue symptoms.

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

Change in severity of narcolepsy symptoms as measured by the Narcolepsy Severity Scale (for participants with NT1) or Narcolepsy Severity Scale-2 (for participants with NT2)

The Narcolepsy Severity Scale is a 15-item participant-reported questionnaire that assesses the severity and consequences of narcolepsy symptoms.

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

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

The British Columbia Cognitive Complaints Inventory is a participant-reported, 6-item, 4-point scale that assesses perceived cognitive difficulties.

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

Change in health-related quality of life as measured by the Short Form-36 physical and mental component summaries

The Short Form-36 includes 36 questions across 8 health domains to measure a participant's functional health and well-being.

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

Change in work productivity as measured by the Work Productivity and Activity Impairment: Narcolepsy work productivity loss score

The Work Productivity and Activity Impairment: Narcolepsy questionnaire is a 6-item scale used to measure impairments over 7 days.

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

Incidence of treatment-emergent adverse events

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.

Time frame: Throughout study (16 months, including Open-label Extension)

Evaluate the pharmacokinetic concentrations of pitolisant and major identified metabolites

Pharmacokinetics is the study of how the body interacts with administered substances for the duration of exposure.

Time frame: Throughout study (16 weeks)