PWS Outcomes Assessment Study

NCT04102839 | Completed

• 1 other identifier: CAS-SOL001-01
• Study Type: observational
• Target: 59
• Locations: 1 country
• Sites: 1

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.

Conditions

Prader-Willi Syndrome

Keywords

Prader-Willi Syndrome; PWS

Outcome Measures

Primary Outcomes (1)

• Meaningful Change in Symptom Categories

  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)

  From baseline for interview #2 (Week 13-16), From baseline for interview #3 (Week 26-28), From baseline for interview #4 (Week 65-67)

Interventions

Interview OTHER

Qualitative Interview

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Caregivers of PWS patients who are participating in Soleno C601 and C602 Clinical Studies.

You may qualify if:

• 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
• 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

You may not qualify if:

• n/a

Sponsors & Collaborators

• Soleno Therapeutics, Inc.: lead
• Casimir Trials: collaborator

Study Sites (1)

Casimir Trials

Plymouth, Massachusetts, 02360, United States

Location

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

Interviews as Topic

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Design

• Study Type: observational
• Observational Model: FAMILY BASED
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 23, 2019
• First Posted: September 25, 2019
• Study Start: July 15, 2019
• Primary Completion: June 8, 2021
• Study Completion: June 8, 2021
• Last Updated: November 4, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)