Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

Fondazione Telethon

Status: Completed

Conditions: Severe Combined Immunodeficiency Due to ADA Deficiency

Treatments: Drug: STRIMVELIS

Study type: Observational

Funder types: Other

Identifiers

NCT03232203

STRIM-001

Details and patient eligibility

About

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.

Enrollment: 16 patients

Sex: All

Ages: 18+ years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

16 participants in 2 patient groups

Health care providers

Description:

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

Treatment:

Drug: STRIMVELIS

Parent/carer

Description:

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

Treatment:

Drug: STRIMVELIS