Severe Combined Immunodeficiency Due to ADA Deficiency
Conditions
Keywords
Human leukocyte antigen, Key Risk Message, ADA-SCID, Health care providers
Brief summary
STRIMVELIS is a medicinal product that restores adenosine deaminase (ADA) function in hematopoietic cell lineages, thereby preventing impaired immune function. STRIMVELIS is indicated for the treatment of patients with ADA- severe combined immunodeficiency (SCID), for whom suitable human leukocyte antigen (HLA)-matched related stem cell donor is not available. The objective of this study is to evaluate the effectiveness of routine and additional risk minimization measures by assessing the understanding of referring health care providers (HCPs) and parents/carers (hereby referred as participants) with regard to the specific risks associated with STRIMVELIS. In this cross-sectional study, surveys will be provided to referring HCPs and parents/carers of children approximately six months after treatment with STRIMVELIS. The study will recruit for approximately two years or until a maximum of 10 referring HCPs and 10 parents/carers have completed their respective surveys, whichever occurs first.
Interventions
DRUGSTRIMVELIS
It is the Autologous cluster of differentiation (CD) 34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA complementary Deoxyribonucleic acid (cDNA) sequence. HCP who have previously referred a patient for STRIMVELIS treatment or a parent's/carer's child who previously received treatment with STRIMVELIS will be recruited to the study
Sponsors
Fondazione Telethon
Study design
Observational model
COHORT
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
* HCPs or HCPs' close family members may not have been employees of Orchard, Pharmaceutical Product Development, LLC (PPD), the Food and Drug Administration (FDA), or the European Medicines Agency (EMA). * HCPs must be licensed * An HCP must not have previously completed a survey regarding STRIMVELIS educational materials. * An HCP must have previously referred a patient for STRIMVELIS treatment. * Parents/carers or parents'/carers' close family members may not have been employees of Orchard, PPD, FDA, or EMA. * A parent/carer must not have previously completed a survey regarding STRIMVELIS educational materials. * A parent's or carer's child must have previously received treatment with STRIMVELIS
Exclusion criteria
* No
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of HCPs providing the correct response | Up to 2 years | A series of questions concerning the specific risks associated with STRIMVELIS will be asked during survey. Data from all survey respondents will be analyzed and reported as descriptive statistics. |
| Proportion of Parents/Carers providing the correct response | Up to 2 years | A series of questions concerning the specific risks associated with STRIMVELIS will be asked during survey. Data from all survey respondents will be analyzed and reported as descriptive statistics. |
Countries
Italy
Outcome results
None listed