Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Group 1 | Approximately 8 subjects with WAS between ages of 12 to 30 years will be included in Group 1. |
| |
| Group 2 | Approximately 8 primary caregivers of subjects with WAS between ages 8 to 30 years will be included in Group 2. |
| |
| Group 3 | Approximately 5 primary caregivers of subjects with WAS under the age of 8 years will be included in Group 3. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Patient Interview guide | Other | The interviewer will conduct the interview for subjects with WAS using a semi-structured patient interview guide. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of subject reported perspectives on WAS | Open-ended interviews will be conducted in adolescent and adult subjects to understand their perspective on WAS. | Up to 90 minutes |
| Number of caregiver reported perspectives on WAS | Open-ended interviews will be conducted in caregivers of subjects with WAS to understand their perspective on WAS. | Up to 90 minutes |
| Number of subject reported preventative measures taken to avoid infection and bleeding | The preventative measures taken to avoid infection and bleeding as reported by subjects during open-ended interviews will be summarized. | Up to 90 minutes |
| Number of caregiver reported preventative measures taken to avoid infection and bleeding | The preventative measures taken to avoid infection and bleeding as reported by caregivers during open-ended interviews will be summarized. | Up to 90 minutes |
| Number of subject reported awareness on the risk of disease | Open-ended interviews will be conducted in adolescent and adult subjects to understand their awareness on the risk of disease (that is, risk of future complications and potential rapid decline). | Up to 90 minutes |
| Number of caregiver reported awareness on the risk of disease | Open-ended interviews will be conducted in caregivers of WAS subjects to understand their awareness on the risk of disease (that is, risk of future complications and potential rapid decline). | Up to 90 minutes |
Not provided
Not provided
Inclusion Criteria:
Subjects:
Caregivers:
Exclusion Criteria:
Subjects
Not provided
A convenience sample of approximately, 21 subjects with WAS and primary caregivers of WAS subjects will be included in the study.
Not provided
| Name | Affiliation | Role |
|---|---|---|
| GSK Clinical Trials | GlaxoSmithKline | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| GSK Investigational Site | Collegeville | Pennsylvania | 19426 | United States |
Not provided
| Label | URL |
|---|---|
| Results for study 208034 can be found on the GSK Clinical Study Register. | View source |
Not provided
Not provided
Not provided
Not provided
Not provided
| Caregiver interview guide | Other | The interviewer will conduct the interview for caregivers of subjects with WAS using a semi-structured caregiver interview guide. |
|
| Sociodemographic questionnaire | Other | At the end of interview, subjects will be required to complete subject or caregiver versions of country-specific sociodemographic questionnaire. |
|
| Pediatric quality of life (PedsQL) questionnaire | Other | 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. |
|
| Clinical questionnaire | Other | At the end of interview, subjects will be required to complete subject or caregiver versions of clinical questionnaire. |
|
| Number of treatment sequences received by subjects | Open-ended interviews will be conducted to understand the treatments received by subjects in terms of sequencing. | Up to 90 minutes |
| Number of subject reported reasons for discontinuation or change in treatment option | Open-ended interviews will be conducted in adolescent and adult subjects to understand reasons for discontinuation or change in treatment option for WAS. | Up to 90 minutes |
| Number of caregiver reported reasons for discontinuation or change in treatment option | Open-ended interviews will be conducted in caregivers of WAS subjects to understand reasons for discontinuation or change in treatment option for WAS. | Up to 90 minutes |
| Analysis of tolerability of treatment by subjects | Open-ended interviews will be conducted in subjects and caregivers to understand the tolerability of WAS treatment by subjects. | Up to 90 minutes |
| Number of subject reported treatment burden | Open-ended interviews will be conducted in adolescent and adult subjects to analyze treatment burden for subjects. | Up to 90 minutes |
| Number of caregiver reported treatment burden | Open-ended interviews will be conducted in caregivers of WAS subjects to analyze treatment burden for caregivers. | Up to 90 minutes |
| Number of subject reported perspectives on the risk associated with treatment | Open-ended interviews will be conducted in adolescent and adult subjects to understand their perspective on the risks associated with each treatment. | Up to 90 minutes |
| Number of caregiver reported perspectives on the risk associated with treatment | Open-ended interviews will be conducted in caregivers of WAS subjects to understand their perspective on the risks associated with each treatment. | Up to 90 minutes |
| Number of key concepts of interest | Open-ended interviews will be conducted to identify the important concepts of interest that may be used in future clinical trials. | Up to 90 minutes |
| ID | Term |
|---|---|
| D014923 | Wiskott-Aldrich Syndrome |
| D007153 | Immunologic Deficiency Syndromes |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D008231 | Lymphopenia |
| D007970 | Leukopenia |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D007960 | Leukocyte Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
| D000081207 | Primary Immunodeficiency Diseases |
| D007154 | Immune System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D011795 | Surveys and Questionnaires |
| ID | Term |
|---|---|
| D003625 | Data Collection |
| D004812 | Epidemiologic Methods |
| D008919 | Investigative Techniques |
| D017531 | Health Care Evaluation Mechanisms |
| D011787 | Quality of Health Care |
| D017530 | Health Care Quality, Access, and Evaluation |
| D011634 | Public Health |
| D004778 | Environment and Public Health |
Not provided
Not provided