NCT03232203

Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

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

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 16 (actual)
• Sponsor: Fondazione Telethon · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Accepted

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.

Conditions

• Severe Combined Immunodeficiency Due to ADA Deficiency

Interventions

Type	Name	Description
DRUG	STRIMVELIS	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

Timeline

• Start date: 2018-04-12
• Primary completion: 2021-06-25
• Completion: 2021-06-25
• First posted: 2017-07-27
• Last updated: 2023-11-14

Locations

1 site across 1 country: Italy