Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome Clinical Trial

Official title:

A Dose Titration Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome With a Double-Blind, Placebo-Controlled, Randomized Withdrawal Extension

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02034071
• Other study ID #: PC025
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: January 8, 2014
• Last updated: August 30, 2016
• Start date: April 2014
• Est. completion date: May 2015

Study information

• Verified date: August 2016
• Source: Essentialis, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

• This is a single-center, open-label, single-arm study with a double-blind, placebo-controlled, randomized withdrawal extension. Patients are initiated on a DCCR dose of about 1.5 mg/kg (maximum starting dose of 145 mg) and are titrated every 14 days to about 2.4 mg/kg, 3.3 mg/kg, 4.2 mg/kg, and 5.1 mg/kg (maximum dose of 507.5 mg). These DCCR doses are equivalent to diazoxide doses of 1.03, 1.66, 2.28, 2.9, and 3.52 mg/kg. The administered dose will be as close to the mg/kg dosing as can be achieved by the available dose strengths of DCCR. Patients will be up-titrated at each visit at the discretion of the investigator. All patients will be continued in the double-blind, placebo-controlled, randomized withdrawal extension. Any patient who showed an increase in resting energy expenditure and/or a reduction in hyperphagia from Baseline through Day 55 or Day 69 will be designated a responder, whereas all others will be designated non-responders. Responders will be randomized in a 1:1 ratio either to continue on active treatment at the dose they were treated with on Day 69 or to the placebo equivalent of that dose for an additional 4 weeks. Non-responders will continue open label treatment during the extension.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 13
• Est. completion date: May 2015
• Est. primary completion date: May 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 10 Years to 22 Years

Eligibility

Inclusion Criteria:

• Children. adolescents and young adults with genetically confirmed Prader-Willi syndrome

• Ages at = 10 years and = 22 years

• Generally healthy as documented by the medical history, physical examination, vital sign assessments, 12-lead electrocardiogram (ECG), and clinical laboratory assessments

• BMI exceeds the 95th percentile of the age specific BMI value on the CDC BMI charts

• Fasting glucose = 126 mg/dL

• HbA1c = 6.5 %

Exclusion Criteria:

• Administration of investigational drugs within 1 month prior to Screening Visit

• Anticipated requirement for use of prohibited medications

• History of allergic reaction or significant intolerance to: diazoxide, thiazides or sulfonamides

• Anticipate transitions in their care from family home to group home or other similar potentially disruptive changes

• Congestive heart failure or known compromised cardiac reserve

• Any other clinically significant endocrine, cardiovascular, pulmonary, neurological, psychiatric, hepatic, gastrointestinal, hematological, renal, or dermatological disease interfering with the assessments of the investigational drug, according to the Investigator

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• DCCR

• Placebo

Locations

Country	Name	City	State
United States	University of California, Irvine	Orange	California

Sponsors (1)

Lead Sponsor	Collaborator
Essentialis, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Weight		Percent Change from Baseline through Day 69	No
Other	Weight		Percent Change from Day 69 through Day 97	No
Other	Resting energy expenditure		Change from Baseline through Day 69	No
Other	Hyperphagia using hyperphagia questionnaire		Change from Baseline through Day 69	No
Other	Percent Body Fat		Change from Baseline through Day 69	No
Other	Percent Body Fat		Change from Day 69 through Day 97	No
Other	Lipids	Percent change from Baseline through Day 69 for triglycerides, total cholesterol, LDL cholesterol, HDL cholesterol, and non-HDL cholesterol	Percent Change from Baseline through Day 69	No
Other	Lipids	Percent change from Day 69 through Day 97 for triglycerides, total cholesterol, LDL cholesterol, HDL cholesterol, and non-HDL cholesterol	Percent Change from Day 69 Through Day 97	No
Primary	Hyperphagia using hyperphagia questionnaire		Change from Day 69 through Day 97	No
Primary	Resting energy expenditure		Change from Day 69 through Day 97	No

External Links

•   Foundation for Prader-Willi Research - About Prader-Willi syndrome
•   Genetics Home Reference - Prader-Willi syndrome
•   Prader-Willi Syndrome Association USA