Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome
Research type
Research Study
Full title
An open-label study of DCCR (diazoxide choline) extended-release tablets in patients with Prader-Willi syndrome (PWS)
IRAS ID
1007921
Contact name
Anish Bhatnagar
Contact email
Sponsor organisation
Soleno Therapeutics UK Ltd
Clinicaltrials.gov Identifier
Research summary
The study is being conducted in patients with Prader-Willi syndrome (PWS) ages 4 years and older. Patients will be eligible to participate if they meet all of the defined inclusion criteria and none of the exclusion criteria. This research study will evaluate the long-term safety and efficacy of DCCR (diazoxide choline) extended-release tablets in patients with PWS. This study will be conducted in the United States and at NHS hospitals in England and Scotland, who have participated in Study C602 and is being paid for by Soleno Therapeutics, Inc (Soleno).
The study duration is up to 5 years (60 months). The first visit will be an in-clinic visit then telephone or telemedicine (video) visits will be performed every 2 weeks during the 6 week titration period. Regular study visits will be performed as telemedicine visits at Month 6 and 12. After the first year, telephone visits may alternate with yearly telemedicine visits if this is more suitable, as long as assessments are completed according to the Schedule of Events (SOE). In-clinic visits may occur as needed and are at the discretion of the Investigator. The in-clinic visit will include safety assessments, collection of blood and urine samples, and completion of questionnaires.REC name
London - City & East Research Ethics Committee
REC reference
23/LO/0543
Date of REC Opinion
4 Aug 2023
REC opinion
Further Information Favourable Opinion