GH-4831: CROITRE Registry :French registry of children treated with Norditropin® for short stature associated with Noonan Syndrome

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Prospective and retrospective design

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  SOMATROPIN
• Medical condition to be studied:
  Noonan syndrome

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)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  221

Study design details

• Main study objective:
  The primary objective is to describe the long-term change of height in children treated with Norditropin® for short stature due to NS over a 4-year period in routine clinical practice in France.
• Outcomes:
  Change in height standard deviation score, According to study protocol section 8.1.1.2
• Data analysis plan:
  The following statistics will be provided for quantitative variables: number of patients with non-missing and missing values, mean, standard deviation, median, first quartile (Q1), third quartile (Q3), minimum and maximum value, mode (for discrete quantitative variables with limited variability). For qualitative variables, the number and proportion of each variable modality will be provided together with the number of missing values. Proportions will be computed based on the population with non-missing data. Missing data will not be imputed or replaced. A descriptive analysis will be conducted on all study endpoints for which data has been collected both on FAS (GAS for primary endpoint) and per subgroups of interest as long as that the sizes of the subgroups are relevant for statistical estimations.