Prospective and retrospective, single-cohort, multicenter observational long-term study in short children born small for gestational age (SGA) after treatment with Saizen® or with other recombinant human growth hormone (r-hGH) products (SALTO)

Study type

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine, other:
  Saizen
• Study drug International non-proprietary name (INN) or common name:
  SOMATROPIN
• Anatomical Therapeutic Chemical (ATC) code:
  (H01AC01) somatropin

  somatropin
• Medical condition to be studied:
  Gestational age test abnormal

Population studied

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

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)
• Estimated number of subjects:
  300

Study design details

• Main study objective:
  To assess the long-term safety of Saizen® or other r-hGH treatment for 10 years after cessation of treatment, in terms of occurrence of type 2 diabetes mellitus and malignancies, in a minimum of 200 subjects born SGA who received Saizen® or other r-hGH products for the treatment of short stature.
• Outcomes:
  The number and proportion of subjects diagnosed at any time during the 10 year follow-up will be tabulated for the two primary endpoints:- Type 2 diabetes mellitus- Any malignancy, - Metabolic syndrome and/or changes in glucose metabolism parameters as per Glycaemia parameters, fasting plasma glucose and postprandial glucose, and/or fasting plasma insulin levels as specified in the protocol.- Malignancies- Correlation of T2DM and/or malignancies will be established against BPI, BMI and others as specified in the protocol.
• Data analysis plan:
  No statistical significance testing will be performed. The study will contribute at the estimation of the incidence of type 2 diabetes mellitus and the incidence of any malignancy among study participants.For primary endpoints, details for each subject of the safety population will be listed and will include at least, time on follow-up, onset date(s) of event(s) and description of event(s).A sensitivity analysis will be performed on the completer population.All secondary endpoints will be analysed using appropriate statistics. For quantitative variables, statistics are the number of non-missing values (N), number of missing values, mean, standard deviation (SD), minimum (Min), first quartile (Q1), median, third quartile (Q3) and maximum (Max). For qualitative variables, statistics within categories are number ofnon-missing values (N), number of missing values and percentages of subjects.