Wiskott-Aldrich SyndromeNCT03399461
Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS)
An observational study of Wiskott-Aldrich Syndrome, run by GlaxoSmithKline at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 12 to 30 years, men only
- Conditions the study is for
- Wiskott-Aldrich Syndrome
- What is being tested?
- Patient Interview guide, Caregiver interview guide and Sociodemographic questionnaire
- How many people
- 19 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Jan 2018to 14 Sep 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 30 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: A convenience sample of approximately, 21 subjects with WAS and primary caregivers of WAS subjects will be included in the study.
Who may be able to join
- 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.
Show all 15Show fewer
- 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.
Who cannot join
- 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 locations and contacts
1 site in 1 country.
United States1 site
GSK Investigational Site
Collegeville, Pennsylvania, 19426
About this study
Official title: 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.
- Conditions
- Wiskott-Aldrich Syndrome
- Being tested
- Patient Interview guide, Caregiver interview guide, Sociodemographic questionnaire, Pediatric quality of life (PedsQL) questionnaire, Clinical questionnaire
- Keywords
- United States, United Kingdom, Wiskott-Aldrich Syndrome, Caregiver, Interview, immune deficiency, France
- Medical subject headings
- Wiskott-Aldrich Syndrome, Immunologic Deficiency Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Patient Interview guideOther
The interviewer will conduct the interview for subjects with WAS using a semi-structured patient interview guide.
Caregiver interview guideOther
The interviewer will conduct the interview for caregivers of subjects with WAS using a semi-structured caregiver interview guide.
Sociodemographic questionnaireOther
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) questionnaireOther
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 questionnaireOther
At the end of interview, subjects will be required to complete subject or caregiver versions of clinical questionnaire.
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Approximately 8 subjects with WAS between ages of 12 to 30 years will be included in Group 1. Receives: Other: Patient Interview guide; Other: Sociodemographic questionnaire; Other: Pediatric quality of life (PedsQL) questionnaire; Other: Clinical questionnaire | |
| Group 2 | Approximately 8 primary caregivers of subjects with WAS between ages 8 to 30 years will be included in Group 2. Receives: Other: Caregiver interview guide; Other: Sociodemographic questionnaire; Other: Pediatric quality of life (PedsQL) questionnaire; Other: Clinical questionnaire | |
| Group 3 | Approximately 5 primary caregivers of subjects with WAS under the age of 8 years will be included in Group 3. Receives: Other: Caregiver interview guide; Other: Sociodemographic questionnaire; Other: Pediatric quality of life (PedsQL) questionnaire; Other: Clinical questionnaire |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subject reported perspectives on WAS
Open-ended interviews will be conducted in adolescent and adult subjects to understand their perspective on WAS.
Measured: Up to 90 minutes
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.
Measured: Up to 90 minutes
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.
Measured: Up to 90 minutes
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.
Measured: Up to 90 minutes
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).
Measured: Up to 90 minutes
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).
Measured: Up to 90 minutes
Number of treatment sequences received by subjects
Open-ended interviews will be conducted to understand the treatments received by subjects in terms of sequencing.
Measured: Up to 90 minutes
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.
Measured: Up to 90 minutes
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.
Measured: Up to 90 minutes
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.
Measured: Up to 90 minutes
Number of subject reported treatment burden
Open-ended interviews will be conducted in adolescent and adult subjects to analyze treatment burden for subjects.
Measured: Up to 90 minutes
Number of caregiver reported treatment burden
Open-ended interviews will be conducted in caregivers of WAS subjects to analyze treatment burden for caregivers.
Measured: Up to 90 minutes
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.
Measured: Up to 90 minutes
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.
Measured: Up to 90 minutes
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.
Measured: Up to 90 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jan 2018
- Study start24 Jan 2018
- Primary completion14 Sep 2018
- Study completion14 Sep 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wiskott-Aldrich SyndromeStudies of the same condition, open and under way.
- More trials run by GlaxoSmithKlineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- GlaxoSmithKline
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- GSK Clinical Trials, Study director, GlaxoSmithKline
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03399461
- Sponsor's protocol ID
- 208034
- Status last verified
- Mar 2019
- First submitted
- 8 Jan 2018
- Last updated
- 14 Mar 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Understanding of the Patient and Caregiver Experience of Wiskott-Aldrich Syndrome (WAS)
- ClinicalTrials.gov ID
- NCT03399461
- Acronym / sponsor ID
- 208034
- Conditions
- Wiskott-Aldrich Syndrome
- Keywords
- United States, United Kingdom, Wiskott-Aldrich Syndrome, Caregiver, Interview, immune deficiency, France
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- GlaxoSmithKlineIndustry (pharma, biotech, devices)
- Study start
- 24 Jan 2018 (actual)
- Primary completion
- 14 Sep 2018 (actual)
- Study completion
- 14 Sep 2018 (actual)
- First posted
- 16 Jan 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group 1 | – | Other: Patient Interview guide, Other: Sociodemographic questionnaire, Other: Pediatric quality of life (PedsQL) questionnaire, Other: Clinical questionnaireApproximately 8 subjects with WAS between ages of 12 to 30 years will be included in Group 1. |
| Group 2 | – | Other: Caregiver interview guide, Other: Sociodemographic questionnaire, Other: Pediatric quality of life (PedsQL) questionnaire, Other: Clinical questionnaireApproximately 8 primary caregivers of subjects with WAS between ages 8 to 30 years will be included in Group 2. |
| Group 3 | – | Other: Caregiver interview guide, Other: Sociodemographic questionnaire, Other: Pediatric quality of life (PedsQL) questionnaire, Other: Clinical questionnaireApproximately 5 primary caregivers of subjects with WAS under the age of 8 years will be included in Group 3. |
Who may qualify
- Ages
- 12 to 30 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- A convenience sample of approximately, 21 subjects with WAS and primary caregivers of WAS subjects will be included in the study.
Key inclusion criteria (15 listed)
- 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.
Key exclusion criteria (4 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Patient Interview guideThe interviewer will conduct the interview for subjects with WAS using a semi-structured patient interview guide. | Other | Group 1 |
| Caregiver interview guideThe interviewer will conduct the interview for caregivers of subjects with WAS using a semi-structured caregiver interview guide. | Other | Group 2, Group 3 |
| Sociodemographic questionnaireAt the end of interview, subjects will be required to complete subject or caregiver versions of country-specific sociodemographic questionnaire. | Other | Group 1, Group 2, Group 3 |
| Pediatric quality of life (PedsQL) questionnaireThe 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 | Group 1, Group 2, Group 3 |
| Clinical questionnaireAt the end of interview, subjects will be required to complete subject or caregiver versions of clinical questionnaire. | Other | Group 1, Group 2, Group 3 |
Primary outcome
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.
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.
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.
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.
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).
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).
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.
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.
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.
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.
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.
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.
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.
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.
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Mar 2019.
- Results for study 208034 can be found on the GSK Clinical Study Register.Link provided by the sponsor.
Related trials for Wiskott-Aldrich Syndrome
Active, not recruiting
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
SCID, Omenn's Syndrome
Enrolling by invitationObservational study
Analysis of the Therapeutic and Clinical Profile of Cannabinoid Treatments: A Real-World Study
Chronic Pain, Depression Disorders
RecruitingObservational study
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
RecruitingPhase 2
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Enrolling by invitationPhase 1/2
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
GSK Clinical Trials study director
GlaxoSmithKline
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Wiskott-Aldrich Syndrome, run by GlaxoSmithKline at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of subject reported perspectives on WAS (time frame: Up to 90 minutes). The study lists 15 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Mar 2019.