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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


Study Design


Related Conditions & MeSH terms


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

See also
  Status Clinical Trial Phase
Withdrawn NCT04140539 - A Clinical Study to Enable Process Validation of Commercial Grade OTL-101 Phase 2/Phase 3
Completed NCT03765632 - Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID Phase 1/Phase 2
Completed NCT02999984 - Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID Phase 1/Phase 2
Active, not recruiting NCT04959890 - Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy