Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)
Public ClinicalTrials.gov record NCT03399461. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Understanding of the Patient and Caregiver Experience of Wiskott-Aldrich Syndrome (WAS)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT03399461
- Recruitment status
- Completed
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 19 participants
Conditions and interventions
Conditions
Interventions
- Patient Interview guide Other
- Caregiver interview guide Other
- Sociodemographic questionnaire Other
- Pediatric quality of life (PedsQL) questionnaire Other
- Clinical questionnaire Other
Other
Eligibility (public fields only)
- Age range
- 12 Years to 30 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 23, 2018
- Primary completion
- Sep 13, 2018
- Completion
- Sep 13, 2018
- Last update posted
- Mar 13, 2019
2018
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| GSK Investigational Site | Collegeville | Pennsylvania | 19426 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03399461, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 13, 2019 · Synced Sep 5, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03399461 live on ClinicalTrials.gov.