Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)

Completed

• Conditions: Wiskott-Aldrich Syndrome
• Interventions: Other: Caregiver interview guide; Other: Patient Interview guide; Other: Sociodemographic questionnaire; Other: Pediatric quality of life (PedsQL) questionnaire; Other: Clinical questionnaire

• Registration Number: NCT03399461
• Lead Sponsor: GlaxoSmithKline

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 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.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2018-01-08
• Study First Submitted QC: 2018-01-08
• Study First Posted: 2018-01-16
• Study Start: 2018-01-24
• Primary Completion: 2018-09-14
• Study Completion: 2018-09-14
• Status Verified: 2019-03
• Last Update Submitted: 2019-03-13
• Last Update Posted: 2019-03-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 19

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.

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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).

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Group 1	Clinical questionnaire	Approximately 8 subjects with WAS between ages of 12 to 30 years will be included in Group 1.
Group 1	Pediatric quality of life (PedsQL) questionnaire	Approximately 8 subjects with WAS between ages of 12 to 30 years will be included in Group 1.
Group 2	Sociodemographic questionnaire	Approximately 8 primary caregivers of subjects with WAS between ages 8 to 30 years will be included in Group 2.
Group 3	Caregiver interview guide	Approximately 5 primary caregivers of subjects with WAS under the age of 8 years will be included in Group 3.
Group 3	Clinical questionnaire	Approximately 5 primary caregivers of subjects with WAS under the age of 8 years will be included in Group 3.
Group 1	Patient Interview guide	Approximately 8 subjects with WAS between ages of 12 to 30 years will be included in Group 1.
Group 1	Sociodemographic questionnaire	Approximately 8 subjects with WAS between ages of 12 to 30 years will be included in Group 1.
Group 2	Caregiver interview guide	Approximately 8 primary caregivers of subjects with WAS between ages 8 to 30 years will be included in Group 2.
Group 2	Pediatric quality of life (PedsQL) questionnaire	Approximately 8 primary caregivers of subjects with WAS between ages 8 to 30 years will be included in Group 2.
Group 3	Pediatric quality of life (PedsQL) questionnaire	Approximately 5 primary caregivers of subjects with WAS under the age of 8 years will be included in Group 3.
Group 2	Clinical questionnaire	Approximately 8 primary caregivers of subjects with WAS between ages 8 to 30 years will be included in Group 2.
Group 3	Sociodemographic questionnaire	Approximately 5 primary caregivers of subjects with WAS under the age of 8 years will be included in Group 3.

Primary Outcome Measures

Name	Time	Method
Number of caregiver reported reasons for discontinuation or change in treatment option	Up to 90 minutes	Open-ended interviews will be conducted in caregivers of WAS subjects to understand reasons for discontinuation or change in treatment option for WAS.
Number of subject reported perspectives on WAS	Up to 90 minutes	Open-ended interviews will be conducted in adolescent and adult subjects to understand their perspective on WAS.
Number of caregiver reported perspectives on WAS	Up to 90 minutes	Open-ended interviews will be conducted in caregivers of subjects with WAS to understand their perspective on WAS.
Number of caregiver reported preventative measures taken to avoid infection and bleeding	Up to 90 minutes	The preventative measures taken to avoid infection and bleeding as reported by caregivers during open-ended interviews will be summarized.
Number of subject reported preventative measures taken to avoid infection and bleeding	Up to 90 minutes	The preventative measures taken to avoid infection and bleeding as reported by subjects during open-ended interviews will be summarized.
Number of subject reported awareness on the risk of disease	Up to 90 minutes	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).
Analysis of tolerability of treatment by subjects	Up to 90 minutes	Open-ended interviews will be conducted in subjects and caregivers to understand the tolerability of WAS treatment by subjects.
Number of caregiver reported awareness on the risk of disease	Up to 90 minutes	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).
Number of treatment sequences received by subjects	Up to 90 minutes	Open-ended interviews will be conducted to understand the treatments received by subjects in terms of sequencing.
Number of subject reported reasons for discontinuation or change in treatment option	Up to 90 minutes	Open-ended interviews will be conducted in adolescent and adult subjects to understand reasons for discontinuation or change in treatment option for WAS.
Number of subject reported treatment burden	Up to 90 minutes	Open-ended interviews will be conducted in adolescent and adult subjects to analyze treatment burden for subjects.
Number of caregiver reported treatment burden	Up to 90 minutes	Open-ended interviews will be conducted in caregivers of WAS subjects to analyze treatment burden for caregivers.
Number of subject reported perspectives on the risk associated with treatment	Up to 90 minutes	Open-ended interviews will be conducted in adolescent and adult subjects to understand their perspective on the risks associated with each treatment.
Number of caregiver reported perspectives on the risk associated with treatment	Up to 90 minutes	Open-ended interviews will be conducted in caregivers of WAS subjects to understand their perspective on the risks associated with each treatment.
Number of key concepts of interest	Up to 90 minutes	Open-ended interviews will be conducted to identify the important concepts of interest that may be used in future clinical trials.

Trial Locations

Locations (1)

GSK Investigational Site; 🇺🇸 Collegeville, Pennsylvania, United States