Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
GlaxoSmithKline
Age
12–30
Sex
MALE
Healthy volunteers
Not accepted
You may be eligible if
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.
You may not be if
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).
