- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03399461
Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)
March 13, 2019 updated by: GlaxoSmithKline
Understanding of the Patient and Caregiver Experience of Wiskott-Aldrich Syndrome (WAS)
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.
Study Overview
Status
Completed
Conditions
Study Type
Observational
Enrollment (Actual)
19
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Pennsylvania
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Collegeville, Pennsylvania, United States, 19426
- GSK Investigational Site
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
12 years to 30 years (Child, Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
Male
Sampling Method
Non-Probability Sample
Study Population
A convenience sample of approximately, 21 subjects with WAS and primary caregivers of WAS subjects will be included in the study.
Description
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).
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
Group 1
Approximately 8 subjects with WAS between ages of 12 to 30 years will be included in Group 1.
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The interviewer will conduct the interview for subjects with WAS using a semi-structured patient 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.
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Group 2
Approximately 8 primary caregivers of subjects with WAS between ages 8 to 30 years will be included in Group 2.
|
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.
The interviewer will conduct the interview for caregivers of subjects with WAS using a semi-structured caregiver interview guide.
|
Group 3
Approximately 5 primary caregivers of subjects with WAS under the age of 8 years will be included in Group 3.
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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.
The interviewer will conduct the interview for caregivers of subjects with WAS using a semi-structured caregiver interview guide.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of subject reported perspectives on WAS
Time Frame: Up to 90 minutes
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Open-ended interviews will be conducted in adolescent and adult subjects to understand their perspective on WAS.
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Up to 90 minutes
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Number of caregiver reported perspectives on WAS
Time Frame: Up to 90 minutes
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Open-ended interviews will be conducted in caregivers of subjects with WAS to understand their perspective on WAS.
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Up to 90 minutes
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Number of subject reported preventative measures taken to avoid infection and bleeding
Time Frame: Up to 90 minutes
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The preventative measures taken to avoid infection and bleeding as reported by subjects during open-ended interviews will be summarized.
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Up to 90 minutes
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Number of caregiver reported preventative measures taken to avoid infection and bleeding
Time Frame: Up to 90 minutes
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The preventative measures taken to avoid infection and bleeding as reported by caregivers during open-ended interviews will be summarized.
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Up to 90 minutes
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Number of subject reported awareness on the risk of disease
Time Frame: Up to 90 minutes
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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).
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Up to 90 minutes
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Number of caregiver reported awareness on the risk of disease
Time Frame: Up to 90 minutes
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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
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Number of treatment sequences received by subjects
Time Frame: Up to 90 minutes
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Open-ended interviews will be conducted to understand the treatments received by subjects in terms of sequencing.
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Up to 90 minutes
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Number of subject reported reasons for discontinuation or change in treatment option
Time Frame: Up to 90 minutes
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Open-ended interviews will be conducted in adolescent and adult subjects to understand reasons for discontinuation or change in treatment option for WAS.
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Up to 90 minutes
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Number of caregiver reported reasons for discontinuation or change in treatment option
Time Frame: Up to 90 minutes
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Open-ended interviews will be conducted in caregivers of WAS subjects to understand reasons for discontinuation or change in treatment option for WAS.
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Up to 90 minutes
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Analysis of tolerability of treatment by subjects
Time Frame: Up to 90 minutes
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Open-ended interviews will be conducted in subjects and caregivers to understand the tolerability of WAS treatment by subjects.
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Up to 90 minutes
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Number of subject reported treatment burden
Time Frame: Up to 90 minutes
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Open-ended interviews will be conducted in adolescent and adult subjects to analyze treatment burden for subjects.
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Up to 90 minutes
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Number of caregiver reported treatment burden
Time Frame: Up to 90 minutes
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Open-ended interviews will be conducted in caregivers of WAS subjects to analyze treatment burden for caregivers.
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Up to 90 minutes
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Number of subject reported perspectives on the risk associated with treatment
Time Frame: Up to 90 minutes
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Open-ended interviews will be conducted in adolescent and adult subjects to understand their perspective on the risks associated with each treatment.
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Up to 90 minutes
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Number of caregiver reported perspectives on the risk associated with treatment
Time Frame: Up to 90 minutes
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Open-ended interviews will be conducted in caregivers of WAS subjects to understand their perspective on the risks associated with each treatment.
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Up to 90 minutes
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Number of key concepts of interest
Time Frame: Up to 90 minutes
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Open-ended interviews will be conducted to identify the important concepts of interest that may be used in future clinical trials.
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Up to 90 minutes
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
January 24, 2018
Primary Completion (Actual)
September 14, 2018
Study Completion (Actual)
September 14, 2018
Study Registration Dates
First Submitted
January 8, 2018
First Submitted That Met QC Criteria
January 8, 2018
First Posted (Actual)
January 16, 2018
Study Record Updates
Last Update Posted (Actual)
March 14, 2019
Last Update Submitted That Met QC Criteria
March 13, 2019
Last Verified
March 1, 2019
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Pathologic Processes
- Immunologic Deficiency Syndromes
- Immune System Diseases
- Disease
- Hematologic Diseases
- Blood Coagulation Disorders, Inherited
- Hemorrhagic Disorders
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Blood Coagulation Disorders
- Leukopenia
- Leukocyte Disorders
- Primary Immunodeficiency Diseases
- Lymphopenia
- Syndrome
- Wiskott-Aldrich Syndrome
Other Study ID Numbers
- 208034
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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