PWS Outcome Assessment Study
Research type
Research Study
Full title
PWS Outcome Assessment Study
IRAS ID
269705
Contact name
Anish Bhatnagar
Contact email
Sponsor organisation
Soleno Therapeutics UK Ltd
Duration of Study in the UK
1 years, 7 months, 2 days
Research summary
Summary of Research
The Prader-Willi syndrome (PWS) Outcomes Assessment study is a longitudinal study designed to conduct remote interviews of caregivers of PWS participants participating in C601 and C602 study sponsored by Soleno. There is no treatment or intervention associated with the study.
During the study, the interviews will be scheduled at four main times:
•between Study C601 Visit 1 (Screening) and Study C601 Visit 3;
•within 3 weeks after the end of Study C601 (within 3 weeks after Study C601, Visit 7);
•within 13 - 15 weeks after enrolment in Study C602 (within 3 weeks after Study C602, Visit 6); and
•within 52 – 54 weeks after enrolment in Study C602 (within 3 weeks after Study C602, Visit 15).
Caregivers of participants in Studies C601 or C602 may enrol in this study at any time during their participation in one of these two studies. If caregivers enrol in this study after Study C601 Visit 3, then their initial interview will be scheduled as soon as possible. They will then complete the interviews for the times that remain.In addition, caregivers will be given the option to collect video data of the Study C601 / C602 PWS participants at the four main times listed above. Also, caregivers will be asked to film “new ability” videos at any time during the study.
All data, including qualitative interviews and daily life videos, will be captured remotely for caregivers and C601 / C602 study PWS participants residing in the United States and the United Kingdom.
Summary of Results
In this sub-study of Soleno Therapeutics’ DESTINY PWS Phase 3 program, interviews were conducted with a semi-structured guide covering specific food-security procedures and three areas of interest: food-related behavior, non-food-related behavior, and aspects of daily life. Caregivers were permitted to consent to participation at any time during the clinical trial, including the open label extension (OLE) phase. Thematic coding for these categories was conducted by trained, blinded researchers. During the interview, caregivers reported whether changes in each domain were positive or negative and if they had a major or minor impact on the patient’s quality of life.
A total of 51 caregivers out of 103 enrolled and completed an Interview #4 at least one year in the open label extension (OLE) study. The mean age of the OLE participants in this sub-study was 11.12 years, including 22 males and 29 females; 6 were in the UK and 45 were in the US. Sixty-seven percent of participants had a deletion subtype and 37% had a non-deletion subtype. The demographic characteristics of participants were similar to that of the DESTINY PWS study population.
After one year in the open label extension (OLE), 33 (64.7%), 30 (58.8%), and 31 (60.8%) caregivers described a positive change in food-related behavior, non-food-related behavior, and daily life, respectively. Of these, 26 (51.0%), 20 (39.2%), and 25 (49.0%), respectively, reported positive change with major impact. Other caregivers reported a positive change with minor impact in the 3 domains (3 [5.9%], 6 [11.8%], and 5 [9.8%], respectively), or did not specify impact (4 [7.8%], 1 [2.0%), 1 [2.0%] respectively).
At the final interview (one year into the OLE period of treatment with DCCR), most caregivers reported positive changes in food-related behaviors, non-food-related behaviors, and/or other aspects of daily life. The qualitative interviews provided insights into participant experiences during the DESTINY PWS clinical trial as seen in the home environment.
REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
19/WM/0289
Date of REC Opinion
12 Nov 2019
REC opinion
Further Information Favourable Opinion