Status:
COMPLETED
Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)
Lead Sponsor:
GlaxoSmithKline
Conditions:
Wiskott-Aldrich Syndrome
Eligibility:
MALE
12-30 years
Brief 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 caregive...
Eligibility Criteria
Inclusion
- 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
- 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).
Key Trial Info
Start Date :
January 24 2018
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
September 14 2018
Estimated Enrollment :
19 Patients enrolled
Trial Details
Trial ID
NCT03399461
Start Date
January 24 2018
End Date
September 14 2018
Last Update
March 14 2019
Active Locations (1)
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1
GSK Investigational Site
Collegeville, Pennsylvania, United States, 19426