Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Registry for Patients Treated with Strimvelis (or GSK2696273) Gene Therapy: Long-Term Prospective, Non-Interventional Follow-up of Safety and Effectiveness (STRIM-003)

Study identification

• EU PAS number:
  EUPAS15795
• Study ID:
  48875
• Official title and acronym:
  Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Registry for Patients Treated with Strimvelis (or GSK2696273) Gene Therapy: Long-Term Prospective, Non-Interventional Follow-up of Safety and Effectiveness (STRIM-003)
• DARWIN EU® study:
  No
• Study countries:
  Austria

  Belgium

  Bulgaria

  Croatia

  Cyprus

  Denmark

  Estonia

  Finland

  France

  Germany

  Greece

  Hungary

  Ireland

  Italy

  Latvia

  Lithuania

  Luxembourg

  Malta

  Netherlands

  Poland

  Portugal

  Romania

  Slovakia

  Slovenia

  Spain

  Sweden

  United Kingdom
• Study description:
  ADA deficiency results in severe combined immunodeficiency (SCID), a fatal autosomal recessive inherited immune disorder. ; Strimvelis™ is a gene therapy that restores ADA function in hematopoietic cell lineages, and in doing so prevents the pathology caused by purine metabolites (i.e., impaired immune function). ; Strimvelis™ is intended for patients with ADA-SCID and for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. ; The objective of this prospective, non-interventional registry is to collect long term safety and effectiveness outcomes for patients that have received Strimvelis™. ; The registry does not have a comparator group and the product will have been given on a single occasion prior to entering this registry. Safety and effectiveness will be assessed for a target number of 50 patients who will have received Strimvelis.™; The registry will close to enrollment when 50 patients have been enrolled but will not close completely until the 50th patient finishes their 15 year follow-up.
• Study status:
  Ongoing

Research institutions and networks

Institutions

IRCCS Ospedale San Raffaele

Study timelines

Date when funding contract was signed

Planned: 07/06/2016

Actual: 07/06/2016

Study start date

Planned: 28/02/2017

Actual: 31/03/2017

Date of interim report, if expected

Planned: 31/03/2021

Actual: 30/03/2021

Date of final study report

Planned: 31/12/2037

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Fondazione Telethon ETS

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 1 (imposed as condition of marketing authorisation)