NCT03399461

Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)

Understanding of the Patient and Caregiver Experience of Wiskott-Aldrich Syndrome (WAS)

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 19 (actual)
• Sponsor: GlaxoSmithKline · Industry
• Sex: Male
• Age: 12 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

WAS is a rare primary immune deficiency disease caused by genetic mutation and is more common in males than females. The purpose of this study is to understand experiences of WAS subjects and caregivers to identify important concepts of interest that could be measured in future Phase IIIb trials. This is a qualitative cross-sectional study that will include a sample of approximately, 8 subjects with WAS and 13 caregivers of subjects with a diagnosis of WAS in the United States, United Kingdom and France. A 60 to 90 minute open-ended interview will be conducted over the telephone or video conference that will be audio-recorded for subsequent transcription. The aim of these interviews is to obtain subject and caregiver perspectives on the impact of WAS and its associated treatments on quality of life and experiences of living with WAS.

Conditions

• Wiskott-Aldrich Syndrome

Interventions

Type	Name	Description
OTHER	Patient Interview guide	The interviewer will conduct the interview for subjects with WAS using a semi-structured patient interview guide.
OTHER	Caregiver interview guide	The interviewer will conduct the interview for caregivers of subjects with WAS using a semi-structured caregiver interview guide.
OTHER	Sociodemographic questionnaire	At the end of interview, subjects will be required to complete subject or caregiver versions of country-specific sociodemographic questionnaire.
OTHER	Pediatric quality of life (PedsQL) questionnaire	The PedsQL is a validated generic measure of health-related quality of life (HRQOL) in children, adolescents, and young adults with acute and chronic health conditions. At the end of interview, subjects will be required to complete subject or caregiver versions of PedsQL questionnaire.
OTHER	Clinical questionnaire	At the end of interview, subjects will be required to complete subject or caregiver versions of clinical questionnaire.

Timeline

• Start date: 2018-01-24
• Primary completion: 2018-09-14
• Completion: 2018-09-14
• First posted: 2018-01-16
• Last updated: 2019-03-14

Locations

1 site across 1 country: United States