Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational Model: Case-Only

Study drug and medical condition

• Name of medicine:
  INCRELEX
• Study drug International non-proprietary name (INN) or common name:
  MECASERMIN
• Anatomical Therapeutic Chemical (ATC) code:
  (H01AC03) mecasermin

  mecasermin
• Medical condition to be studied:
  Severe primary insulin like growth factor-1 deficiency

  Growth failure

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)
• Estimated number of subjects:
  500

Study design details

• Main study objective:
  To collect, analyse and report safety data during and at least 5 years after the end of the treatment in children and adolescents receiving Increlex therapy for SPIGFD according to the locally approved product information.
• Outcomes:
  Incidence of: serious adverse events (SAEs) including any AESI of neoplasia, all targeted adverse events (AEs), all AEs, deaths and withdrawals due to AEs during Increlex® treatment period up to 30 days after the last dose. Changes from baseline for effectiveness variables, Estimation of difference between predicted & final adult height, Modelling effectiveness parameters, Dose administered & duration of exposure, Biological assessments, genetic tests, Incidence of safety events at 2 and 5 years post-treatment, Description of neoplasias (benign and malignant) and hypoglycaemias, Evolution of QoL using EQ-5D-Y.
• Data analysis plan:
  The statistical analyses will be performed in accordance with ICH E9 guideline and will be based on the pooled data from the individual study sites, unless otherwise stated. ; Primary analyses during treatment period: ; - description and incidence of any serious adverse events including neoplasia, ; - incidence of all targeted adverse events ; - description and incidence of all adverse events.