An Open-Label, Long-Term Safety Evaluation Study with DCCR
Research type
Research Study
Full title
An Open-Label, Long-Term Safety Evaluation of Diazoxide Choline Controlled-Release Tablet in Patients with Prader-Willi Syndrome
IRAS ID
268966
Contact name
Anish Bhatnagar
Contact email
Sponsor organisation
Soleno Therapeutics UK Limited
Eudract number
2018-004216-22
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
42702, CPMS - Adoption into CRN
Duration of Study in the UK
1 years, 6 months, 14 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, even after eating a meal), learning disabilities, growth hormone deficiency/short stature, behavioral difficulties, and many other symptoms. Only growth hormone has been approved for use in PWS patients. There are no approved treatments for hyperphagia or other common symptoms many PWS patients have. This research study will evaluate the long-term safety of DCCR (diazoxide choline controlled-release) in PWS patients who have successfully completed the C601 clinical study. This study will be conducted at NHS hospitals in England and Scotland and is being paid for Soleno Therapeutics, Inc (Soleno).
This study involves 16 study visits over approximately 54 weeks. Ten visits will be conducted in clinic at which the participant and the caregiver shall attend. Six visits will be conducted over the telephone. The participant shall fast for at least 8 hours prior to all clinic visits and until blood is taken, the waist is measured, and the participant is weighed. At each clinic 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: DEXA 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/0422
Date of REC Opinion
25 Sep 2019
REC opinion
Further Information Favourable Opinion