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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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 Wit

Brief 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.

Detailed Summary:
Sponsor: Essentialis, Inc.

Current Primary Outcome:

  • Hyperphagia using hyperphagia questionnaire [ Time Frame: Change from Day 69 through Day 97 ]
  • Resting energy expenditure [ Time Frame: Change from Day 69 through Day 97 ]


Original Primary Outcome: Dose of DCCR [ Time Frame: Day 83 ]

The dose of DCCR on Day 83 is defined as the maximally tolerated dose for purposes of the study


Current Secondary Outcome:

Original Secondary Outcome:

  • Hyperphagia using Hyperphagia questionnaire [ Time Frame: Change from baseline to Day 83 ]
  • Hyperphagia using Hyperphagia questionnaire [ Time Frame: Change from Day 83 to Day 111 ]
  • Resting energy expenditure [ Time Frame: Change from baseline to Day 83 ]
  • Resting energy expenditure [ Time Frame: Change from Day 83 to Day 111 ]


Information By: Essentialis, Inc.

Dates:
Date Received: January 8, 2014
Date Started: April 2014
Date Completion:
Last Updated: August 30, 2016
Last Verified: August 2016