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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Health care providers | A HCP survey instrument of approximately 20 questions will be provided. Survey questions will be based on the STRIMVELIS summary of product characteristics and educational materials |
| |
| Parent/carer | A parent/carer survey instrument of approximately 20 questions will be provided. Survey questions will be based on the STRIMVELIS Patient Information Leaflet and educational materials |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| STRIMVELIS | Drug | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of HCPs providing the correct response | 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. | Up to 2 years |
| Proportion of Parents/Carers providing the correct response | 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. | Up to 2 years |
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Inclusion Criteria:
Exclusion Criteria:
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Parents/carers and referring HCPs of patients enrolled within this registry or patients treated with STRIMVELIS outside of the registry will be contacted separately and asked to participate in their respective surveys evaluating the effectiveness of risk minimization measures.
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| Name | Affiliation | Role |
|---|---|---|
| Fondazione Telethon | Fondazione Telethon | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) | Milan | 20132 | Italy |
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| ID | Term |
|---|---|
| C531816 | Severe combined immunodeficiency due to adenosine deaminase deficiency |
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