Wiskott-Aldrich Syndrome Clinical Trial
Official title:
Understanding of the Patient and Caregiver Experience of Wiskott-Aldrich Syndrome (WAS)
NCT number | NCT03399461 |
Other study ID # | 208034 |
Secondary ID | |
Status | Completed |
Phase | |
First received | |
Last updated | |
Start date | January 24, 2018 |
Est. completion date | September 14, 2018 |
Verified date | March 2019 |
Source | GlaxoSmithKline |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
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). |
Country | Name | City | State |
---|---|---|---|
United States | GSK Investigational Site | Collegeville | Pennsylvania |
Lead Sponsor | Collaborator |
---|---|
GlaxoSmithKline |
United States,
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 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT01652092 -
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
|
N/A | |
Completed |
NCT01953016 -
Participation in a Research Registry for Immune Disorders
|
||
Active, not recruiting |
NCT02333760 -
Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
|
Phase 1/Phase 2 | |
Terminated |
NCT01319851 -
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
|
N/A | |
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
Recruiting |
NCT04371939 -
Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome
|
Phase 2 | |
Completed |
NCT01347346 -
Gene Therapy for WAS
|
Phase 1/Phase 2 | |
Completed |
NCT00160355 -
Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
|
Phase 1 | |
Recruiting |
NCT01821781 -
Immune Disorder HSCT Protocol
|
Phase 2 | |
Completed |
NCT01289847 -
A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency
|
Phase 4 | |
Completed |
NCT01347242 -
Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
|
Phase 1/Phase 2 | |
Terminated |
NCT00006054 -
Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies
|
N/A | |
Enrolling by invitation |
NCT03198195 -
Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome
|
N/A | |
Recruiting |
NCT03019809 -
A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
|
Phase 2 | |
Terminated |
NCT00909363 -
Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
|
Phase 2 | |
Enrolling by invitation |
NCT01852370 -
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
|
Phase 1/Phase 2 | |
Completed |
NCT03513328 -
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT01410825 -
Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT00004341 -
Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders
|
N/A | |
Active, not recruiting |
NCT03837483 -
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome
|
Phase 3 |