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

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

Detailed Description
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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.

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

Conditions
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Idiopathic Hypersomnia

Intervention / Treatment
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* Drug: Pentetrazol (PTZ)
* Drug: Placebo oral capsule


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Subject Receives BTD001 first<br> | Drug: Pentetrazol (PTZ)<br>* BTD-001 is an oral capsule of PTZ.<br>* Other names: PTZ;Drug: Placebo oral capsule<br>* Placebo will be presented as oral capsule.<br>|
| Experimental: Subject Receives Placebo first<br> | Drug: Pentetrazol (PTZ)<br>* BTD-001 is an oral capsule of PTZ.<br>* Other names: PTZ;Drug: Placebo oral capsule<br>* Placebo will be presented as oral capsule.<br>|

What is the study measuring?
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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. |