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

Wiskott-Aldrich Syndrome Clinical Trial

Official title:

Understanding of the Patient and Caregiver Experience of Wiskott-Aldrich Syndrome (WAS)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03399461
• Other study ID #: 208034
• Status: Completed
• Start date: January 24, 2018
• Est. completion date: September 14, 2018

Study information

• Verified date: March 2019
• Source: GlaxoSmithKline
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 19
• Est. completion date: September 14, 2018
• Est. primary completion date: September 14, 2018
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years to 30 Years

Eligibility

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).

Related Conditions & MeSH terms

• Syndrome
• Wiskott-Aldrich Syndrome

Intervention

Other:
• Patient Interview guide
The interviewer will conduct the interview for subjects with WAS using a semi-structured patient interview guide.
• Caregiver interview guide
The interviewer will conduct the interview for caregivers of subjects with WAS using a semi-structured caregiver interview guide.
• Sociodemographic questionnaire
At the end of interview, subjects will be required to complete subject or caregiver versions of country-specific sociodemographic questionnaire.
• Pediatric quality of life (PedsQL) 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.
• Clinical questionnaire
At the end of interview, subjects will be required to complete subject or caregiver versions of clinical questionnaire.

Locations

Country	Name	City	State
United States	GSK Investigational Site	Collegeville	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
GlaxoSmithKline

Country where clinical trial is conducted

United States, 

Outcome

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

External Links

•   Results for study 208034 can be found on the GSK Clinical Study Register.