DESTINY PWS - A Study of DCCR in Patients with Prader-Willi Syndrome
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients with Prader-Willi Syndrome
IRAS ID
259342
Contact name
Anish Bhatnagar
Contact email
Sponsor organisation
Soleno Therapeutics UK Limited
Eudract number
2018-004215-50
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
41242, CPMS
Duration of Study in the UK
0 years, 7 months, 30 days
Research summary
The study is being conducted in patients with Prader-Willi syndrome (PWS) ages 4 years and older. PWS is a rare genetic disorder in which patients have hyperphagia (patient always feels hungry and never feels full, even after eating a meal, and will continuously eat if not prevented from eating by a third party), severe obesity, learning disabilities and behavioral difficulties, growth hormone deficiency/short stature and other hormone abnormalities such as abnormal pubertal development, and many other symptoms. So far, only growth hormone has been approved for use in PWS patients which helps to reduce the weight increase. There are no approved treatments for hyperphagia or other common symptoms many PWS patients have. This research study will evaluate if DCCR (diazoxide choline controlled-release) has an effect on hyperphagia and symptoms of PWS syndrome compared to a placebo. (A placebo looks like the study medication being tested, but does not have the active ingredient). Hyperphagia is measured using a validated questionnaire (HC-QT). This study will be conducted at NHS hospitals in England and Scotland. This study is being paid for by Soleno Therapeutics, Inc (Soleno).
This study involves 7 study visits over approximately 15 weeks at which the participant and the caregiver shall attend. The participant shall fast for at least 8 hours prior to these visits and until blood is taken, the waist circumference is measured, and the participant is weighed. At each visit, patients will have typical medical tests and measurements, which will include the following: a physical exam, vital signs, urine pregnancy test for females of child-bearing potential, an interview with the caregiver regarding the participant’s suicidal ideation and behaviour, study drug administration as well as side effect assessment and review of medications taken. At some visits, the following will be performed: DNA sample collection, DXA scan, ECG, and caregiver completion of questionnaires.
The participant will take his/her medication every morning, report any changes in medications to the study team after starting the study, report any change in the participant’s health to the study team, and follow the instructions of the study doctor and study team.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
19/SC/0122
Date of REC Opinion
27 Mar 2019
REC opinion
Favourable Opinion