A 5 Treatment Period Pharmacokinetic Study Evaluating Dose Proportionality and Food Effects of Diazoxide Choline Controlled-Release Tablet (DCCR)

Phase 2

• Conditions: Prader-Willi Syndrome
• Interventions: Drug: Diazoxide choline controlled-release tablet

• Registration Number: NCT02893618
• Lead Sponsor: Essentialis, Inc.

Brief Summary

Open label, parallel-group, single site, 5 treatment-period study with 4 dose levels of DCCR, 1 of which is administered both with and without food.

Detailed Description

Open label, parallel-group, single site, 5 treatment-period study with 4 dose levels of DCCR, 1 of which is administered both with and without food, with a 10 Day washout period between treatments. There will be 8 treatment sequences with 4 subjects randomized to each.

Study Timeline

• Study First Submitted: 2016-08-30
• Status Verified: 2016-09
• Study First Submitted QC: 2016-09-02
• Last Update Submitted: 2016-09-07
• Study First Posted: 2016-09-08
• Last Update Posted: 2016-09-09
• Study Start: 2017-07
• Primary Completion: 2017-10
• Study Completion: 2017-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 32

Inclusion Criteria

• Ability to follow verbal and written instructions
• Informed consent form signed by the subject
• Completed screening within 7 days prior to dosing
• BMI between 18.5 and 35 kg/m2
• Generally healthy
• fasting glucose less than or equal to 100 mg/dL
• HbA1c less than or equal to 6%

Exclusion Criteria

• Pregnancy or breast feeding
• absence of contraception
• administration of investigational drug within 1 month prior to screening
• anticipated requirement for prohibited medication (systemic corticosteroids or anti-diabetic medications)
• allergic reaction to or significant intolerance of diazoxide, thiazides or sulfonamides
• known type 1 or type 2 diabetes mellitus
• congestive heart failure
• gastric bypass surgery
• history of drug or alcohol abuse

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
DCCR 150 mg fasted	Diazoxide choline controlled-release tablet	Administered a single 150 mg dose of DCCR after an overnight fast followed by 4 hours of fasting
DCCR 300 mg fasted	Diazoxide choline controlled-release tablet	Administered a single 300 mg dose of DCCR after an overnight fast followed by 4 hours of fasting
DCCR 300 mg fed	Diazoxide choline controlled-release tablet	Administered a single 300 mg dose of DCCR after a standardized meal
DCCR 75 mg fasted	Diazoxide choline controlled-release tablet	Administered a single 75 mg dose of DCCR after an overnight fast followed by 4 hours of fasting
DCCR 450 mg fasted	Diazoxide choline controlled-release tablet	Administered a single 450 mg dose of DCCR after an overnight fast followed by 4 hours of fasting

Primary Outcome Measures

Name	Time	Method
Pharmacokinetic parameters: Cmax	up to 24 hours	Cmax derived from PK samples taken at 0, 1, 3, 6, 9, 12, 15, 18, 21 and 24 hours post-dose
Pharmacokinetic parameters: AUC0-24	up to 24 hours	AUC0-24 derived from PK samples taken at 0, 1, 3, 6, 9, 12, 15, 18, 21 and 24 hours

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetic parameters: CL/F	up to 24 hours	CL/F derived from PK samples taken at 0, 1, 3, 6, 9, 12, 15, 18, 21 and 24 hours
Pharmacokinetic parameters: Tmax	up to 24 hours	Tmax derived from PK samples taken at 0, 1, 3, 6, 9, 12, 15, 18, 21 and 24 hours