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

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Brief Title

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

Official Title

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

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.
    



Study Type

Observational


Primary Outcome

Number of subject reported perspectives on WAS


Condition

Wiskott-Aldrich Syndrome

Intervention

Patient Interview guide

Study Arms / Comparison Groups

 Group 1
Description:  Approximately 8 subjects with WAS between ages of 12 to 30 years will be included in Group 1.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Other

Estimated Enrollment

19

Start Date

January 24, 2018

Completion Date

September 14, 2018

Primary Completion Date

September 14, 2018

Eligibility 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).
      

Gender

Male

Ages

12 Years - 30 Years

Accepts Healthy Volunteers

No

Contacts

GSK Clinical Trials, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03399461

Organization ID

208034


Responsible Party

Sponsor

Study Sponsor

GlaxoSmithKline


Study Sponsor

GSK Clinical Trials, Study Director, GlaxoSmithKline


Verification Date

March 2019