NCT03232203

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Apr 2018

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 25, 2017

Completed
2 days until next milestone

First Posted

Study publicly available on registry

July 27, 2017

Completed
9 months until next milestone

Study Start

First participant enrolled

April 12, 2018

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 25, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 25, 2021

Completed
Last Updated

November 14, 2023

Status Verified

November 1, 2023

Enrollment Period

3.2 years

First QC Date

July 25, 2017

Last Update Submit

November 10, 2023

Conditions

Severe Combined Immunodeficiency Due to ADA Deficiency

Keywords

Human leukocyte antigenKey Risk MessageADA-SCIDHealth care providers

Outcome Measures

Primary Outcomes (2)

  • 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

Study Arms (2)

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

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

Interventions

STRIMVELISDRUG

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

Health care providersParent/carer

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodProbability 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.

You may qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

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

Milan, 20132, Italy

Location

MeSH Terms

Conditions

Severe combined immunodeficiency due to adenosine deaminase deficiency

Study Officials

  • Fondazione Telethon

    Fondazione Telethon

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
CROSS SECTIONAL
Target Duration
2 Years
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 25, 2017

First Posted

July 27, 2017

Study Start

April 12, 2018

Primary Completion

June 25, 2021

Study Completion

June 25, 2021

Last Updated

November 14, 2023

Record last verified: 2023-11

Locations

IT(1)