A Study of Safety and Efficacy of BTD-001 in Treatment of Patients With Idiopathic Hypersomnia (IH) or Narcolepsy Type 2

A Randomized, Placebo-Controlled, Double-blind, Fixed-Dose, Multiple Cohort, Multiple Crossover, Dose-Finding Study of Oral BTD-001 in Adults With Idiopathic Hypersomnia (IH) or Narcolepsy Type 2

This is a randomized, placebo-controlled, double-blind, multiple cohort, fixed-dose multiple crossover, dose-finding study of oral BTD-001 in adult patients with IH or Narcolepsy without cataplexy (Type 2).

Study Overview

• Status: Completed
• Conditions: Idiopathic Hypersomnia; Narcolepsy Without Cataplexy
• Intervention / Treatment: Drug: Placebo; Drug: BTD-001

• Study Type: Interventional
• Enrollment (Anticipated): 120
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35213
      • Sleep Disorders Center of Alabama
  • California
    • Palo Alto, California, United States, 94304
      • Stanford Medicine Sleep Center
    • San Diego, California, United States, 92103
      • Pacific Research Network
    • Upland, California, United States, 91786
      • Empire Clinical Research
  • Colorado
    • Boulder, Colorado, United States, 80301
      • REM Sleep Medicine, Colorado Sleep Institute
  • Florida
    • Brandon, Florida, United States, 33511
      • PAB Clinical Research
    • Saint Petersburg, Florida, United States, 33707
      • Clinical Research Group of St. Petersburg
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • NeuroTrials Research Inc
    • Atlanta, Georgia, United States, 30329
      • Emory University School of Medicine Emory Sleep Center
    • Macon, Georgia, United States, 31201
      • SleepMed of Central
  • Kentucky
    • Louisville, Kentucky, United States, 40218
      • Kentucky Research Group,
  • Maryland
    • Chevy Chase, Maryland, United States, 20815
      • The Center for Sleep and Wake Disorders
  • Massachusetts
    • Newton, Massachusetts, United States, 02459
      • NeuroCare Center for Sleep
  • Minnesota
    • Maplewood, Minnesota, United States, 55109
      • Neurological Associate
  • Missouri
    • Saint Louis, Missouri, United States, 63143
      • Clayton Sleep Institute
  • New York
    • New York, New York, United States, 10032
      • Columbia University Medical Center
  • North Carolina
    • Raleigh, North Carolina, United States, 27612
      • Wake Research Associates
  • Ohio
    • Cincinnati, Ohio, United States, 45255
      • Community Research Management Assoc.
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73112
      • Lynn Institute
  • South Carolina
    • Columbia, South Carolina, United States, 29201
      • SleepMed of South Carolina
  • Texas
    • Austin, Texas, United States, 78731
      • Future Search Trials of Neurology
    • Houston, Texas, United States, 77063
      • Houston Sleep Center
  • Washington
    • Seattle, Washington, United States, 98122
      • Swedish Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Meets ICSD-3 criteria for IH or Narcolepsy Type 2 and not undergoing pharmacologic treatment for the condition
• Usual nightly total sleep at least 6 hours as single major rest period without naps
• Epworth Sleepiness Scale of 10 or greater
• Males or females age 18 to 65 years

Exclusion Criteria:

• Any disorder causing hypersomnia other than IH or Narcolepsy Type 2
• Usual bedtime later than midnight
• Seizure disorder or history of syncope, unexplained loss of consciousness or seizure in the past 3 years
• Beck Depression Inventory score greater than 19
• Beck Anxiety Inventory score greater than 15
• Significant history of or current suicidal ideation or behavior
• BMI less than 18 kg/m2 or greater than 39 kg/m2
• Positive toxicology screen or breathalyzer test
• Clinically significant abnormal findings on safety assessments
• Any significant medical or psychiatric disease or any condition that would put the patient at risk by participating in the study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Placebo	Drug: Placebo
Experimental: BTD-001	Drug: BTD-001

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy Epworth Sleepiness Scale	Epworth Sleepiness Scale	After 14 days per treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy Maintenance of Wakefulness Test	Maintenance of Wakefulness Test	After 14 days per treatment

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetics (Cmax)	Pharmacokinetic parameters after multiple doses. -Maximum serum concentration (Cmax)	Selected Days 14, 35, and 56
Pharmacokinetics (Tmax)	Pharmacokinetic parameters after multiple doses. -Time of maximum plasma concentration (Tmax)	Selected Days 14, 35, and 56
Pharmacokinetics (T½)	Pharmacokinetic parameters after multiple doses. -Elimination half life (T½)	Selected Days 1, 14, 31, 35, 42, and 56
Pharmacokinetics (AUC)	Pharmacokinetic parameters after multiple doses. -Area under the concentration time curve (AUC)	Selected Days 1, 14, 21, 35, 42, and 56

Collaborators and Investigators

• Sponsor: Balance Therapeutics
• Investigators: Study Director: Lyndon Lien, PhD, Balance Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2015
• Primary Completion (Actual): February 1, 2018
• Study Completion (Actual): February 1, 2018

Study Registration Dates

• First Submitted: July 23, 2015
• First Submitted That Met QC Criteria: July 29, 2015
• First Posted (Estimate): July 31, 2015

Study Record Updates

• Last Update Posted (Actual): January 16, 2019
• Last Update Submitted That Met QC Criteria: January 14, 2019
• Last Verified: January 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Nervous System Diseases; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Disorders of Excessive Somnolence; Narcolepsy; Cataplexy; Idiopathic Hypersomnia
• Other Study ID Numbers: BTD-001 IH201