Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

Severe Combined Immunodeficiency Due to ADA Deficiency Clinical Trial

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

Status Completed

Clinical Trial 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.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• Severe Combined Immunodeficiency
• Severe Combined Immunodeficiency Due to ADA Deficiency

• NCT number: NCT03232203
• Study type: Observational [Patient Registry]
• Source: Fondazione Telethon
• Status: Completed
• Start date: April 12, 2018
• Completion date: June 25, 2021