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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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

  • United States
    • Pennsylvania
      • Collegeville, Pennsylvania, United States, 19426
        • GSK Investigational Site

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Group 1
Approximately 8 subjects with WAS between ages of 12 to 30 years will be included in Group 1.
Other: Patient Interview guide
The interviewer will conduct the interview for subjects with WAS using a semi-structured patient interview guide.
Other: Sociodemographic questionnaire
At the end of interview, subjects will be required to complete subject or caregiver versions of country-specific sociodemographic questionnaire.
Other: 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.
Other: Clinical questionnaire
At the end of interview, subjects will be required to complete subject or caregiver versions of clinical questionnaire.
Group 2
Approximately 8 primary caregivers of subjects with WAS between ages 8 to 30 years will be included in Group 2.
Other: Sociodemographic questionnaire
At the end of interview, subjects will be required to complete subject or caregiver versions of country-specific sociodemographic questionnaire.
Other: 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.
Other: Clinical questionnaire
At the end of interview, subjects will be required to complete subject or caregiver versions of clinical questionnaire.
Other: Caregiver interview guide
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.
Other: Sociodemographic questionnaire
At the end of interview, subjects will be required to complete subject or caregiver versions of country-specific sociodemographic questionnaire.
Other: 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.
Other: Clinical questionnaire
At the end of interview, subjects will be required to complete subject or caregiver versions of clinical questionnaire.
Other: Caregiver interview guide
The interviewer will conduct the interview for caregivers of subjects with WAS using a semi-structured caregiver interview guide.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of subject reported perspectives on WAS
Time Frame: Up to 90 minutes
Open-ended interviews will be conducted in adolescent and adult subjects to understand their perspective on WAS.
Up to 90 minutes
Number of caregiver reported perspectives on WAS
Time Frame: Up to 90 minutes
Open-ended interviews will be conducted in caregivers of subjects with WAS to understand their perspective on WAS.
Up to 90 minutes
Number of subject reported preventative measures taken to avoid infection and bleeding
Time Frame: Up to 90 minutes
The preventative measures taken to avoid infection and bleeding as reported by subjects during open-ended interviews will be summarized.
Up to 90 minutes
Number of caregiver reported preventative measures taken to avoid infection and bleeding
Time Frame: Up to 90 minutes
The preventative measures taken to avoid infection and bleeding as reported by caregivers during open-ended interviews will be summarized.
Up to 90 minutes
Number of subject reported awareness on the risk of disease
Time Frame: 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).
Up to 90 minutes
Number of caregiver reported awareness on the risk of disease
Time Frame: 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).
Up to 90 minutes
Number of treatment sequences received by subjects
Time Frame: Up to 90 minutes
Open-ended interviews will be conducted to understand the treatments received by subjects in terms of sequencing.
Up to 90 minutes
Number of subject reported reasons for discontinuation or change in treatment option
Time Frame: 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.
Up to 90 minutes
Number of caregiver reported reasons for discontinuation or change in treatment option
Time Frame: 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.
Up to 90 minutes
Analysis of tolerability of treatment by subjects
Time Frame: Up to 90 minutes
Open-ended interviews will be conducted in subjects and caregivers to understand the tolerability of WAS treatment by subjects.
Up to 90 minutes
Number of subject reported treatment burden
Time Frame: Up to 90 minutes
Open-ended interviews will be conducted in adolescent and adult subjects to analyze treatment burden for subjects.
Up to 90 minutes
Number of caregiver reported treatment burden
Time Frame: Up to 90 minutes
Open-ended interviews will be conducted in caregivers of WAS subjects to analyze treatment burden for caregivers.
Up to 90 minutes
Number of subject reported perspectives on the risk associated with treatment
Time Frame: 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.
Up to 90 minutes
Number of caregiver reported perspectives on the risk associated with treatment
Time Frame: 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.
Up to 90 minutes
Number of key concepts of interest
Time Frame: 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.
Up to 90 minutes

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
GlaxoSmithKline

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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 24, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 14, 2018

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 14, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 8, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 8, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 16, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 14, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 13, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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