Based on the review of data from more than 50% of patients enrolled, the Data Safety Monitoring Board has recommended continuing a phase III clinical trial of DCCR for PWS. The trial, called DESTINY PWS, is a randomized, double-blind, placebo-controlled study of once daily oral administration of DCCR versus placebo by Soleno Therapeutics, Inc.
Role of the Data Safety Monitoring Board
When a large clinical trial is conducted, data is often monitored and reviewed by a Data Safety Monitoring Board. This board is an independent group of experts who periodically review and evaluate the study data for participant safety, study conduct and progress, and when appropriate, efficacy. The monitoring board will make recommendations on whether to continue, modify or terminate a clinical trial, depending on how the data looks.
This was the second planned Data Safety Monitoring Board Review of the phase III DESTINY PWS clinical trial. Both meetings resulted in the board recommending the continuation without modification of the trial. The outcome of this second meeting was based on the review of data from more than 50% of patients enrolled and treated.
DESTINY PWS Clinical Trial Background
DESTINY PWS is a Phase III, randomized, double-blind, placebo-controlled study of once daily oral administration of DCCR versus placebo in approximately 100 patients with a confirmed diagnosis of PWS. The primary endpoint is change from baseline hyperphagia score at week 13. Patients who complete DESTINY PWS have the option to enroll into C602, the open-label safety extension study.
In July 2018, the U.S. Food and Drug Administration designated the investigation of DCCR for the treatment of PWS to be a Fast Track development program. Prior to this, diazoxide choline received orphan designation for the treatment of PWS in the U.S. and in the E.U.
About Soleno Therapeutics
Soleno Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases. “We are pleased to again receive the DSMB’s recommendation to continue the Phase III trial as planned and without modification, which provides ongoing support of DCCR’s safety profile,” said Dr. Anish Bhatnagar, Chief Executive Officer of Soleno. “Enrollment in DESTINY PWS remains on track for the availability of top-line data in the first half of 2020.”