A Study of Oral BTD-001 in Adults With Idiopathic Hypersomnia (ARISE2)

A Randomized, Placebo-Controlled, Double-Blind, Crossover Study of Oral BTD-001 in Adults With Idiopathic Hypersomnia

This is a randomized, placebo-controlled, double-blind, crossover study of oral BTD-001 in adults with Idiopathic Hypersomnia.

Study Overview

• Status: Unknown
• Conditions: Idiopathic Hypersomnia
• Intervention / Treatment: Drug: Pentetrazol (PTZ); Drug: Placebo oral capsule

Detailed Description

This is a randomized, placebo-controlled, double-blind, crossover study of oral BTD-001 in adults with Idiopathic Hypersomnia (IH). The objectives of this study are two-fold: 1) To evaluate the efficacy of BTD-001 in subjects with IH as reflected by changes in cognitive, sleep, functional, and quality-of-life measures and 2) To characterize the safety and tolerability of oral BTD-001 administered to subjects with IH.

• Study Type: Interventional
• Enrollment (Anticipated): 80
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35213
      • Sleep Disorders Center of Alabama
  • Arizona
    • Scottsdale, Arizona, United States, 85054
      • Mayo Clinic Hospital
  • California
    • Orange, California, United States, 92868
      • SDS Clinical Trials
    • San Diego, California, United States, 92128
      • Pacific Research Network
    • San Ramon, California, United States, 94583
      • Sleep Medicine Specialists of California
    • Santa Monica, California, United States, 90404
      • Santa Monica Clinical Trials
  • Colorado
    • Boulder, Colorado, United States, 80301
      • REM Sleep Medicine
  • Florida
    • Brandon, Florida, United States, 33511
      • PAB Clinical Research
    • Miami, Florida, United States, 33126
      • Sleep Medicine Specialists of South Florida
    • Winter Park, Florida, United States, 32789
      • Florida Premier Research Institute
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • NeuroTrials Research Inc.
    • Macon, Georgia, United States, 31210
      • Sleep Practicioners
  • Illinois
    • Northbrook, Illinois, United States, 60062
      • Northshore Sleep Medicine
  • Indiana
    • Fort Wayne, Indiana, United States, 46804
      • Fort Wayne Neurology
  • Kansas
    • Lenexa, Kansas, United States, 66214
      • Rowe Neurology Institute
  • 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
  • Michigan
    • Sterling Heights, Michigan, United States, 48314
      • Sleep and Attention Disorders Institute
  • Minnesota
    • Saint Paul, Minnesota, United States, 55130
      • Neuroscience Center
  • Missouri
    • Chesterfield, Missouri, United States, 63017
      • St. Luke's Sleep Medicine and Research Center
  • New York
    • New York, New York, United States, 10467
      • Albert Einstein College of Medicine
    • New York, New York, United States, 10032
      • Columbia University - Department of Neurology
  • North Carolina
    • Huntersville, North Carolina, United States, 28078
      • Research Carolina of Huntersville
  • Ohio
    • Cincinnati, Ohio, United States, 95242
      • Intrepid Research
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73112
      • Lynn Health Science Institute
  • Pennsylvania
    • Danville, Pennsylvania, United States, 17822
      • Geisinger
    • Monroeville, Pennsylvania, United States, 15146
      • Consolidated Clinical Trials
  • South Carolina
    • Columbia, South Carolina, United States, 29201
      • Bogan Sleep Consultants, LLC
  • Texas
    • Austin, Texas, United States, 78731
      • Future Search Trials
    • 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 70 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Males or Females age 18 to 70 years old
• Onset of hypersomnia between age 10 and 30 years of age
• An Epworth Sleepiness Scale score of greater than or equal to 11
• Able to comply with requirements for concomitant medications and consumption of caffeine, alcohol, tobacco and nicotine (if on stimulants, agree to adhere to stable regimen throughout the trial)
• Females with a negative pregnancy test AND who are non-lactating
• Sexually active females of childbearing potential must be willing to use a highly effective method of birth control
• Sexually active males must have a vasectomy or use condoms

Exclusion Criteria:

• History of any disorder causing hypersomnia other than IH
• Evidence of circadian-rhythm disorder
• Sleep apnea syndrome
• Use of CPAP
• Obese subjects with BMI greater than or equal to 35kg/m2
• History of or current seizure disorder or history of syncope, unexplained loss of consciousnesses or seizure in the past 3 years as well as any past history of benzodiazepine and/or barbiturate and/or alcohol-related withdrawal seizures
• Columbia-Suicide Severity Rating Scale (C-SSRS) findings consistent with significant history of or current suicidal ideation or behavior
• Subjects who fail to wash out medications for IH or any other prohibited medications
• Positive toxicology screen test during the Screening or Baseline Visits.
• Clinically significant abnormal findings from physical, electrocardiogram (ECG) or laboratory assessments at Screening
• History of or current significant pulmonary, cardiac, neurological or psychiatric disease, substance dependence, porphyria, malignancy (with exception of local cutaneous squamous or basal cell carinomas or local cervical squamous cell carcinoma resolved after resection) or hypothyroidism (unless euthyroid at Screening Visit and treated with a stable dose of medication for at least 3 months prior to the Baseline Visit).
• Participation in a clinical drug trial within 4 weeks of Screening Visit

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: CROSSOVER
• Masking: SINGLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Subject Receives BTD001 first	Drug: Pentetrazol (PTZ); BTD-001 is an oral capsule of PTZ.; Other Names: PTZ; Drug: Placebo oral capsule; Placebo will be presented as oral capsule.
EXPERIMENTAL: Subject Receives Placebo first	Drug: Pentetrazol (PTZ); BTD-001 is an oral capsule of PTZ.; Other Names: PTZ; Drug: Placebo oral capsule; Placebo will be presented as oral capsule.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The efficacy of BTD-001 in subjects with IH as reflected by the IH Symptom Diary	Measurements: IH Symptom Diary.	Change from baseline.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The efficacy of BTD-001 in subjects with IH as reflected by changes in functional measures.	Measurements: ESS (Epworth Sleepiness Scale).	Change from baseline.
The efficacy of BTD-001 in subjects with IH as reflected by changes in sleep measures.	Measurements: MWT (Maintenance of Wakefulness Test)	Change from baseline.

Collaborators and Investigators

• Sponsor: Balance Therapeutics
• Investigators: Study Director: Morgan Lam, Chief Operating Officer

Study record dates

Study Major Dates

• Study Start (ACTUAL): May 29, 2018
• Primary Completion (ANTICIPATED): May 1, 2020
• Study Completion (ANTICIPATED): May 28, 2020

Study Registration Dates

• First Submitted: May 1, 2018
• First Submitted That Met QC Criteria: May 18, 2018
• First Posted (ACTUAL): May 31, 2018

Study Record Updates

• Last Update Posted (ACTUAL): April 29, 2020
• Last Update Submitted That Met QC Criteria: April 28, 2020
• Last Verified: April 1, 2020

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; Idiopathic Hypersomnia; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; GABA Agents; Central Nervous System Stimulants; GABA Antagonists; Convulsants; Pentylenetetrazole
• Other Study ID Numbers: BTD-001 IH202

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No