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

NCT03399461 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 19

Last updated 2019-03-14

No results posted yet for this study

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

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.

Sponsors & Collaborators

Principal Investigators

  • GSK Clinical Trials · GlaxoSmithKline

Eligibility

Min Age
12 Years
Max Age
30 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-01-24
Primary Completion
2018-09-14
Completion
2018-09-14

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03399461 on ClinicalTrials.gov