Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
Status:
Completed
Trial end date:
2015-05-01
Target enrollment:
Participant gender:
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.