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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03232203
• Other study ID #: STRIM-001
• Status: Completed
• Start date: April 12, 2018
• Est. completion date: June 25, 2021

Study information

• Verified date: November 2023
• Source: Fondazione Telethon
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: June 25, 2021
• Est. primary completion date: June 25, 2021
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

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

Locations

Country	Name	City	State
Italy	Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)	Milan

Sponsors (1)

Lead Sponsor	Collaborator
Fondazione Telethon

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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