NCT03714373

Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period

An Open-Label, Long-Term Safety and Efficacy Evaluation of Diazoxide Choline Extended-Release Tablets in Participants With Prader-Willi Syndrome With a Double-Blind, Placebo-Controlled, Randomized Withdrawal Period

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 115 (actual)
• Sponsor: Soleno Therapeutics, Inc. · Industry
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

Summary

This is a multi-center, multi-period study with an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period evaluating the safety and efficacy of DCCR treatment.

Detailed description

115 PWS participants who completed clinical study C601 will be enrolled into the OLE Period. All participants in the Open Label Extension (OLE) Period will receive open-label DCCR. The actual number of participants eligible to enroll in the double-blind, placebo-controlled, randomized withdrawal (RW) period will be limited to those participants taking DCCR in the OLE Period at the time of the RW Period Visit 1 (Baseline/Randomization Visit).The treatment groups in the C602 RW Period are those participants randomized to receive DCCR and those participants randomized to receive Placebo. Participants will be randomized in a 1:1 ratio (DCCR:Placebo).

Conditions

• Prader-Willi Syndrome

Interventions

Type	Name	Description
DRUG	DCCR	Once daily oral administration of open-label DCCR tablet(s) during the OLE Period
DRUG	DCCR	Once daily oral administration of double-blind (DCCR) tablet(s) during the RW Period
DRUG	Placebo for DCCR	Once daily oral administration of double-blind (placebo for DCCR) tablet(s) during the RW Period

Timeline

• Start date: 2018-10-01
• Primary completion: 2023-08-17
• Completion: 2023-08-17
• First posted: 2018-10-22
• Last updated: 2024-04-19

Locations

28 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study