The Data Safety Monitoring Board (DSMB) has recommended, for the second time, the continuation of Soleno Therapeutics’ Phase 3 DESTINY PWS clinical trial evaluating the effects of diazoxide choline controlled release (DCCR) for treating children and adults with Prader-Willi syndrome (PWS).
DCCR is an oral, extended-release salt formulation of diazoxide that works by preventing the release of two neuropeptides — neuropeptide Y (NPY) and agouti-related protein (AgRP) — that are thought to trigger excessive appetite and food consumption (hyperphagia) in patients with PWS.
DESTINY PWS (NCT03440814) is a 13-week, randomized, double-blind, placebo-controlled, Phase 3 trial designed to evaluate the effects of DCCR when administered orally, once-a-day, at a dose of 75–450 mg to approximately 100 patients with a confirmed diagnosis of PWS, compared to a placebo.
The primary goal of the trial, which is still recruiting participants, is to evaluate changes in the scores of a hyperphagia questionnaire (a survey that measures patients’ hyperphagia-related behaviors) from baseline to week 13. Secondary goals include assessing changes from baseline to week 13 in patients’ body fat mass, and in clinical progress estimated by clinicians and caregivers.
After completing the 13-week treatment period of DESTINY PWS, patients will have the opportunity to enroll in C602, the trial’s nine-month, open-label safety extension study, to continue treatment with DCCR.
The DSMB is an independent group of experts, including physicians, statisticians, and patient advocates, who are responsible for monitoring the safety of patients participating in trials. The DSMB may decide to allow the trial to continue without issuing any recommendations, to modify certain aspects of its clinical design, or to terminate it altogether due to safety concerns.
After reviewing safety information from more than half of the patients participating in DESTINY PWS, the DSMB decided the study should continue without any alterations during its second planned meeting.
“We are pleased to again receive the DSMB’s recommendation to continue the Phase 3 trial as planned and without modification, which provides ongoing support of DCCR’s safety profile,” Anish Bhatnagar, CEO of Soleno, said in a press release.
“Enrollment in DESTINY PWS remains on track for the availability of top-line data in the first half of 2020,” Bhatnagar said.
In July 2018, the U.S. Food and Drug Administration (FDA) granted fast track status to DCCR to accelerate its development. Diazoxide choline already had received the designation of orphan drug for the treatment of PWS in the U.S. and European Union.