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 type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study

Non-interventional study

• Non-interventional study design, other:
  Registry

Study drug and medical condition

• Medicinal product name:
  STRIMVELIS
• Medical condition to be studied:
  Adenosine deaminase increased

Population studied

• Age groups:
  • Preterm newborn infants (0 – 27 days)
  • Term newborn infants (0 – 27 days)
  • Infants and toddlers (28 days – 23 months)
  • Children (2 to < 12 years)
  • Adolescents (12 to < 18 years)
  • Adults (18 to < 46 years)
• Estimated number of subjects:
  50

Study design details

• Outcomes:
  Assessment of: Effectiveness: Survival, Treatments of interest, Immune reconstitution, metabolite detoxification, Vector copy number, severe infections, non-immunological manifestations, Pediatric development and PRO Safety: Reported AEs and SAEs, Laboratory blood test results, Fertility/pregnancy related outcomes, RIS analysis and replication competent retrovirus.
• Data analysis plan:
  This is an exposure registry without a comparison group and no inferential hypothesis testing will be performed. All data, including patient demographics, laboratory values, and AE/SAE rates will be summarized using descriptive statistics.