Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

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

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.

Study Overview

• Status: Completed
• Conditions: Severe Combined Immunodeficiency Due to ADA Deficiency
• Intervention / Treatment: Drug: STRIMVELIS

• Study Type: Observational
• Enrollment (Actual): 16

Contacts and Locations

Study Locations

• Italy
  • Milan, Italy, 20132
    • Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

• Sampling Method: Probability Sample

Study Population

Parents/carers and referring HCPs of patients enrolled within this registry or patients treated with STRIMVELIS outside of the registry will be contacted separately and asked to participate in their respective surveys evaluating the effectiveness of risk minimization measures.

Description

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 Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Health care providers; 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; 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
Parent/carer; 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; 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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Fondazione Telethon
• Investigators: Study Director: Fondazione Telethon, Fondazione Telethon

Study record dates

Study Major Dates

• Study Start (Actual): April 12, 2018
• Primary Completion (Actual): June 25, 2021
• Study Completion (Actual): June 25, 2021

Study Registration Dates

• First Submitted: July 25, 2017
• First Submitted That Met QC Criteria: July 25, 2017
• First Posted (Actual): July 27, 2017

Study Record Updates

• Last Update Posted (Estimated): November 14, 2023
• Last Update Submitted That Met QC Criteria: November 10, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Keywords: ADA-SCID; Human leukocyte antigen; Health care providers; Key Risk Message
• Additional Relevant MeSH Terms: Metabolic Diseases; Immunologic Deficiency Syndromes; Immune System Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; DNA Repair-Deficiency Disorders; Primary Immunodeficiency Diseases; Severe Combined Immunodeficiency
• Other Study ID Numbers: STRIM-001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No