Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

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.

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Key information

Conditions

Severe Combined Immunodeficiency Due to ADA Deficiency Severe combined immunodeficiency due to adenosine deaminase deficiency

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)

Milan, 20132, Italy

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

STRIMVELIS

Drug

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

Primary outcomes

Proportion of HCPs providing the correct response

Time frame: 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

Time frame: 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.

Sponsors and collaborators

Lead sponsor

Fondazione Telethon

Other

Registry information

Official study title

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

Important dates

Study start: 2018
Primary completion: 2021
Study completion: 2021
First posted: Jul 27, 2017
Registry last updated: Nov 14, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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Completed