Last updated: 03/13/2019 15:30:12

Targeted literature review and subject interviews in Wiskott-Aldrich Syndrome (WAS)

GSK study ID
208034
See study documents
Clinicaltrials.gov ID
NCT03399461
EudraCT ID
Not applicable
EU CT Number
Not applicable
Trial status
Study complete
Study complete
Overview
Eligibility
Locations
Study documents
Results summary
Plain language summaries
Additional information

Trial overview

Official title: Understanding of the Patient and Caregiver Experience of Wiskott-Aldrich Syndrome (WAS)
Trial description: 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.
Primary purpose:
Not applicable
Trial design:
Not applicable
Masking:
Not applicable
Allocation:
Not applicable
Primary outcomes:

Number of subject reported perspectives on WAS

Timeframe: Up to 90 minutes

Number of caregiver reported perspectives on WAS

Timeframe: Up to 90 minutes

Number of subject reported preventative measures taken to avoid infection and bleeding

Timeframe: Up to 90 minutes

Number of caregiver reported preventative measures taken to avoid infection and bleeding

Timeframe: Up to 90 minutes

Number of subject reported awareness on the risk of disease

Timeframe: Up to 90 minutes

Number of caregiver reported awareness on the risk of disease

Timeframe: Up to 90 minutes

Number of treatment sequences received by subjects

Timeframe: Up to 90 minutes

Number of subject reported reasons for discontinuation or change in treatment option

Timeframe: Up to 90 minutes

Number of caregiver reported reasons for discontinuation or change in treatment option

Timeframe: Up to 90 minutes

Analysis of tolerability of treatment by subjects

Timeframe: Up to 90 minutes

Number of subject reported treatment burden

Timeframe: Up to 90 minutes

Number of caregiver reported treatment burden

Timeframe: Up to 90 minutes

Number of subject reported perspectives on the risk associated with treatment

Timeframe: Up to 90 minutes

Number of caregiver reported perspectives on the risk associated with treatment

Timeframe: Up to 90 minutes

Number of key concepts of interest

Timeframe: Up to 90 minutes

Secondary outcomes:
Not applicable
Interventions:
  • Other: Patient Interview guide
  • Other: Caregiver interview guide
  • Other: Sociodemographic questionnaire
  • Other: Pediatric quality of life (PedsQL) questionnaire
  • Other: Clinical questionnaire
    • Enrollment:
    19
    Primary completion date:
    2018-14-09
    Observational study model:
    Cohort
    Time perspective:
    Cross-Sectional
    Clinical publications:
    Not applicable
    Medical condition
    Wiskott-Aldrich Syndrome
    Product
    GSK2696275
    Collaborators
    Not applicable
    Study date(s)
    January 2018 to September 2018
    Type
    Observational
    Phase
    Not applicable

    Participation criteria

    Sex
    Male
    Age
    12 - 30 years
    Accepts healthy volunteers
    No
    • Subjects:
    • Male adolescents or young adults ranging in age from 12 through 30 years old.
    • Subjects
    • Subjects who have previously received gene therapy treatment
    Inclusion and exclusion criteria
    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).

    Trial location(s)

    Location
    Status
    Contact us
    Contact us
    Location
    GSK Investigational Site
    Collegeville, Pennsylvania, United States, 19426
    Status
    Study Complete
    Contact us

    Study documents

    Scientific result summary
    Available language(s): English
    Download document(s)
    Protocol
    Available language(s): English
    Download document(s)

    If you wish to request for full study report, please contact - [email protected]

    Results overview

    Refer to study documents

    Recruitment status
    Study complete
    Actual primary completion date
    2018-14-09
    Actual study completion date
    2018-14-09

    Plain language summaries

    Not applicable. GSK’s transparency policy provides for Plain Language Summaries for Interventional studies.

    Additional information about the trial

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