Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

Completed

• Conditions: Severe Combined Immunodeficiency Due to ADA Deficiency
• Interventions: Drug: STRIMVELIS

• Registration Number: NCT03232203
• Lead Sponsor: Fondazione Telethon

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-07-25
• Study First Submitted QC: 2017-07-25
• Study First Posted: 2017-07-27
• Study Start: 2018-04-12
• Primary Completion: 2021-06-25
• Study Completion: 2021-06-25
• Status Verified: 2023-11
• Last Update Submitted: 2023-11-10
• Last Update Posted: 2023-11-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 16

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Health care providers	STRIMVELIS	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	STRIMVELIS	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

Primary Outcome Measures

Name	Time	Method
Proportion of HCPs providing the correct response	Up to 2 years	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.
Proportion of Parents/Carers providing the correct response	Up to 2 years	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.

Trial Locations

Locations (1)

Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET); 🇮🇹 Milan, Italy