Last updated: 03/13/2019 15:30:12
Targeted literature review and subject interviews in Wiskott-Aldrich Syndrome (WAS)
Clinicaltrials.gov ID
EudraCT ID
Not applicable
EU CT Number
Not applicable
Trial status
Finalized
Finalized
Trial overview
Official title: Understanding of the Patient and Caregiver Experience of Wiskott-Aldrich Syndrome (WAS)
Trial description: 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.
Primary purpose:
Not applicable
Trial design:
Not applicable
Masking:
Not applicable
Allocation:
Not applicable
Primary outcomes:
Number of subject reported perspectives on WAS
Timeframe: Up to 90 minutes
Number of caregiver reported perspectives on WAS
Timeframe: Up to 90 minutes
Number of subject reported preventative measures taken to avoid infection and bleeding
Timeframe: Up to 90 minutes
Number of caregiver reported preventative measures taken to avoid infection and bleeding
Timeframe: Up to 90 minutes
Number of subject reported awareness on the risk of disease
Timeframe: Up to 90 minutes
Number of caregiver reported awareness on the risk of disease
Timeframe: Up to 90 minutes
Number of treatment sequences received by subjects
Timeframe: Up to 90 minutes
Number of subject reported reasons for discontinuation or change in treatment option
Timeframe: Up to 90 minutes
Number of caregiver reported reasons for discontinuation or change in treatment option
Timeframe: Up to 90 minutes
Analysis of tolerability of treatment by subjects
Timeframe: Up to 90 minutes
Number of subject reported treatment burden
Timeframe: Up to 90 minutes
Number of caregiver reported treatment burden
Timeframe: Up to 90 minutes
Number of subject reported perspectives on the risk associated with treatment
Timeframe: Up to 90 minutes
Number of caregiver reported perspectives on the risk associated with treatment
Timeframe: Up to 90 minutes
Number of key concepts of interest
Timeframe: Up to 90 minutes
Secondary outcomes:
Not applicable
Interventions:
Other: Patient Interview guide
Other: Caregiver interview guide
Other: Sociodemographic questionnaire
Other: Pediatric quality of life (PedsQL) questionnaire
Other: Clinical questionnaire
Enrollment:
19
Observational study model:
Cohort
Primary completion date:
2018-14-09
Time perspective:
Cross-Sectional
Clinical publications:
Not applicable
Medical condition
Wiskott-Aldrich Syndrome
Product
GSK2696275
Collaborators
Not applicable
Study date(s)
January 2018 to September 2018
Type
Observational
Phase
Not applicable
Participation criteria
Sex
Male
Age
12 - 30 years
Accepts healthy volunteers
No
- Subjects:
- Male adolescents or young adults ranging in age from 12 through 30 years old.
- Subjects
- Subjects who have previously received gene therapy treatment
Inclusion and exclusion criteria
Inclusion criteria:
- Subjects:
- Male adolescents or young adults ranging in age from 12 through 30 years old.
- Diagnosis of WAS defined by WAS genetic mutation with a clinical score >=1 or self-reported disease severity.
- Subjects who are “conservatively managed” or have not received treatment for WAS; a maximum of 5 subjects who have received a hematopoietic stem cell transplant within the preceding two years.
- Able to read, speak, and understand English or French sufficiently to complete all assessments.
- Willing and able to participate in an audio-recorded, telephone or video conference session, including adherence to the interview instructions and completion of all questionnaires.
- Parent/guardian/patient signed informed consent. Caregivers:
- Caregivers of children >=2 years of age diagnosed with WAS; at least 5 caregivers of subjects under the age of 8.
- Caregivers of subjects with a diagnosis of WAS defined by WAS genetic mutation with a clinical score >=1 or self-reported disease severity.
- Caregivers of subjects who are “conservatively managed” or have not received treatment for WAS; At least 2 caregivers of subjects who have received a hematopoietic stem cell transplant within the preceding two years.
- Caregivers are defined as primary caregiver.
- Able to read, speak, and understand English or French sufficiently to complete all assessments.
- Willing and able to participate in an audio-recorded, telephone or video conferencing interview session, including adherence to the interview instructions and completion of all questionnaires.
- Caregivers of Wiskott-Aldrich patients can be of any age or gender and do not have to follow the patient inclusion criteria, as long as their child/patient meets the inclusion criteria, as outlined.
Exclusion criteria:
- Subjects
- Subjects who have previously received gene therapy treatment Caregivers
- Caregivers of subjects who have previously received gene therapy treatment
- Professional caregivers (that is, at home nurse or equivalent).
Trial location(s)
Location
GSK Investigational Site
Collegeville, Pennsylvania, United States, 19426
Status
Study Complete
Study documents
Scientific result summary
Available language(s): English
Protocol
Available language(s): English
If you wish to request for full study report, please contact - [email protected]
Results overview
Refer to study documents
Recruitment status
Finalized
Actual primary completion date
2018-14-09
Actual study completion date
2018-14-09
Plain language summaries
Not applicable. GSK’s transparency policy provides for Plain Language Summaries for Interventional studies.
Additional information about the trial
Participate in clinical trial
Additional information
Results for study 208034 can be found on the GSK Clinical Study Register.
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