PWS Outcomes Assessment Study

Completed

• Conditions: Prader-Willi Syndrome

• Registration Number: NCT04102839
• Lead Sponsor: Soleno Therapeutics, Inc.

Brief Summary

This is a longitudinal study during which qualitative interviews will be conducted with caregivers of Soleno C601/C602 study subjects. There is an additional option for caregivers to collect video data of PWS patients doing specific activities of daily life.

The purpose of this study is to understand the real-world and nuanced impact of a potential therapeutic on individual PWS patients. The results of this study will complement the outcomes being captured during the Soleno C601/C602 clinical studies.

There is no treatment or intervention associated with this study.

Detailed Description

Not available

Study Timeline

• Study Start: 2019-07-15
• Study First Submitted: 2019-08-23
• Study First Submitted QC: 2019-09-23
• Study First Posted: 2019-09-25
• Primary Completion: 2021-06-08
• Study Completion: 2021-06-08
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-31
• Last Update Posted: 2024-11-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 59

Inclusion Criteria

Key Inclusion Criteria for Interviews:

Eligible caregivers must:

• Be willing and able to provide informed consent in English
• Care for a C601/C602 trial subject or potential trial subject
• Have access to a smartphone or the internet
• Be able to converse in English

Key Inclusion Criteria for Video Capture:

Eligible PWS participants for the optional daily life video capture must:

• Be willing and able to provide informed consent or assent.
• Be enrolled in the C601/C602 trial
• Be able to converse in English
• Have a caregiver who has access to an Apple or Android smartphone or has internet access and is willing to use a wi-fi-only device to record and upload videos
• Have a caregiver who is willing and able to record activity videos

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Exclusion Criteria

• n/a

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Meaningful Change in Symptom Categories	From baseline for interview #2 (Week 13-16), From baseline for interview #3 (Week 26-28), From baseline for interview #4 (Week 65-67)	The percentage of caregivers reporting meaningful change in each of the symptom categories in the Study C601 treated arm versus the Study C601 placebo arm as measured by the five-point quantification scale anchored in the qualitative questions (Scale: 1=very meaningful negative change, 2=slightly meaningful negative change, 3=no meaningful change, 4=slightly meaningful change, 5=very meaningful positive change)

Trial Locations

Locations (1)

Casimir Trials; 🇺🇸 Plymouth, Massachusetts, United States