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

• Sponsor: Balance Therapeutics
• Information provided by (Responsible Party): Balance Therapeutics

Study Description

Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Idiopathic Hypersomnia	Drug: Pentetrazol (PTZ); Drug: Placebo oral capsule	Phase 2

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 Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 80 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: Single (Investigator)
• Primary Purpose: Treatment
• Official Title: A Randomized, Placebo-Controlled, Double-Blind, Crossover Study of Oral BTD-001 in Adults With Idiopathic Hypersomnia
• Actual Study Start Date: May 29, 2018
• Estimated Primary Completion Date: September 2019
• Estimated Study Completion Date: December 2019

Arms and Interventions

Arm	Intervention/treatment
Experimental: Subject Receives BTD001 first	Drug: Pentetrazol (PTZ); BTD-001 is an oral capsule of PTZ.; Other Name: 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 Name: PTZ; Drug: Placebo oral capsule; Placebo will be presented as oral capsule.

Outcome Measures

Primary Outcome Measures :

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

Secondary Outcome Measures :

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

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 70 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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

Contacts and Locations

Contacts

Contact: Nichole Baio; 650-351-7677; nichole.baio@balance-therapeutics.com

  Show 32 Study Locations

Sponsors and Collaborators

Balance Therapeutics

Investigators

• Study Director: Morgan Lam; Chief Operating Officer

More Information

• Responsible Party: Balance Therapeutics
• ClinicalTrials.gov Identifier: NCT03542851 History of Changes
• Other Study ID Numbers: BTD-001 IH202
• First Posted: May 31, 2018
• Last Update Posted: July 23, 2019
• Last Verified: July 2019

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Hypersomnolence, Idiopathic; Disorders of Excessive Somnolence; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Nervous System Diseases; Mental Disorders; Pentylenetetrazole; Convulsants; Central Nervous System Stimulants; Physiological Effects of Drugs; GABA Antagonists; GABA Agents; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action