Clinical trial NCT03232203: Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)
Official title: Evaluation of Referring HCPs' and Parents'/Carers' Understanding of Specific Risks Associated With Strimvelis™ Treatment
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Severe Combined Immunodeficiency Due to ADA Deficiency
Keywords: Human leukocyte antigen
Key Risk Message
ADA-SCID
Health care providers
Lead sponsor: Fondazione Telethon
Sponsor class: OTHER
First posted: 2017-07-27
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
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.

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.


Eligibility criteria: 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

Primary outcomes:
measure: Proportion of HCPs providing the correct response
timeFrame: Up to 2 years
measure: Proportion of Parents/Carers providing the correct response
timeFrame: Up to 2 years
Secondary outcomes:
Other outcomes:
Locations:
city: Milan
country: Italy
facility: Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Study officials:
affiliation: Fondazione Telethon
name: Fondazione Telethon
role: STUDY_DIRECTOR
References: