Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

Study Overview
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Brief Summary
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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.

Official Title
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Evaluation of Referring HCPs' and Parents'/Carers' Understanding of Specific Risks Associated With Strimvelis™ Treatment

Conditions
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Severe Combined Immunodeficiency Due to ADA Deficiency

Intervention / Treatment
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* Drug: STRIMVELIS


Participation Criteria
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Eligibility Criteria
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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 exclusion criteria

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Health care providers<br>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 | Drug: STRIMVELIS<br>* 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<br>|
| Parent/carer<br>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 | Drug: STRIMVELIS<br>* 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<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Human leukocyte antigen, Key Risk Message, ADA-SCID, Health care providers