Status:

COMPLETED

Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

Lead Sponsor:

Fondazione Telethon

Conditions:

Severe Combined Immunodeficiency Due to ADA Deficiency

Eligibility:

All Genders

18+ years

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 treatmen...

Eligibility Criteria

Inclusion

  • 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

  • No exclusion criteria

Key Trial Info

Start Date :

April 12 2018

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

June 25 2021

Estimated Enrollment :

16 Patients enrolled

Trial Details

Trial ID

NCT03232203

Start Date

April 12 2018

End Date

June 25 2021

Last Update

November 14 2023

Active Locations (1)

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

Milan, Italy, 20132