An observational study in Wiskott-Aldrich Syndrome, sponsored by GlaxoSmithKline. Completed at 1 site in United States. Open to male participants aged 12 Years to 30 Years. Per ClinicalTrials.gov, last updated 2019-03-14.
Sponsored by GlaxoSmithKline · Observational
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.
37 studies on the registry are indexed under Wiskott-Aldrich Syndrome; 4 are open to participants now.
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A convenience sample of approximately, 21 subjects with WAS and primary caregivers of WAS subjects will be included in the study.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Subjects:
Caregivers:
Exclusion Criteria:
Subjects
Approximately 8 subjects with WAS between ages of 12 to 30 years will be included in Group 1.
Other: Patient Interview guide · Other: Sociodemographic questionnaire · Other: Pediatric quality of life (PedsQL) questionnaire · Other: Clinical questionnaire
Approximately 8 primary caregivers of subjects with WAS between ages 8 to 30 years will be included in Group 2.
Other: Caregiver interview guide · Other: Sociodemographic questionnaire · Other: Pediatric quality of life (PedsQL) questionnaire · Other: Clinical questionnaire
Approximately 5 primary caregivers of subjects with WAS under the age of 8 years will be included in Group 3.
Other: Caregiver interview guide · Other: Sociodemographic questionnaire · Other: Pediatric quality of life (PedsQL) questionnaire · Other: Clinical questionnaire
The interviewer will conduct the interview for subjects with WAS using a semi-structured patient interview guide.
The interviewer will conduct the interview for caregivers of subjects with WAS using a semi-structured caregiver interview guide.
At the end of interview, subjects will be required to complete subject or caregiver versions of country-specific sociodemographic 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.
At the end of interview, subjects will be required to complete subject or caregiver versions of clinical questionnaire.
Number of subject reported perspectives on WAS
Open-ended interviews will be conducted in adolescent and adult subjects to understand their perspective on WAS.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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).
Time frame: 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).
Time frame: Up to 90 minutes
Number of treatment sequences received by subjects
Open-ended interviews will be conducted to understand the treatments received by subjects in terms of sequencing.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Up to 90 minutes
This study is completed, as verified in Mar 2019. You cannot join it, but the record below documents what was studied.
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