Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Change in height standard deviation score |
Measured in standard deviation scores (SDS) ranging from -10 to +10 |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Number of adverse drug reactions (ADR) |
Count of events |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Number of serious adverse drug reactions (SADR) |
Count of events |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Number of serious adverse events (SAE) |
Count of events |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Number of adverse events (AE) |
Count of events |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Education |
Number of participants is measured under the categorical variables - standard education, education with assistance and educational measures |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
School level |
Number of participants is measured under the variables - preschool, elementary school, secondary school and high school |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Educational measures |
Number of participants is measured under the categorical variables - physiotherapy, psychomotricity, occupational therapy and speech therapy |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Tanner stage |
Measured as units on scale. 3 sub-scales used - Pubic hair scale, Female breast development scale and Male external genitalia scale. Tanner stages range from Stage 1 to 5 with Tanner Stage 1 corresponding to the pre-pubertal form and Tanner Stage 5 corresponding to the final adult form |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in pulse rate |
Measured in beats per minute |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in diastolic blood pressure |
Measured in millimeters of Mercury (mm Hg) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in systolic blood pressure |
Measured in mm Hg |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in corrected QT-interval (Electrocardiogram) |
Measured in millisecond (msec) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in echocardiogram parameters (mm) |
Change in Left ventricular interventricular septal maximum thickness in diastole and Left ventricular end diastolic diameter is measured in millimeter (mm) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in echocardiogram parameters (%) |
Change in Left Ventricular Fractional Shortening (LVFS) and Left Ventricular Ejection Fraction (LVEF) is measured in percentage (%) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in echocardiogram parameters (cm/s) |
Change in Diastolic function (Ea and Aa) measured in centimeters per second (cm/s) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in complete blood count (CBC) (g/dL) |
Change in Haemoglobin (Hb) measured in grams per deciLiter (g/dL) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in CBC (G/L) |
Change in white blood cells, lymphocytes, monocytes and platelets measured in grams per liter (G/L) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in Insulin-like growth factor 1 (IGF-1) level (ng/ml) |
Measured in nanograms per milliliter (ng/ml) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in IGF-1 level (SDS) |
Measured in SDS |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in Haemoglobin A1c (HbA1c) level |
Measured in % |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in fasting insulin level |
Measured in milligrams per deciliter (mg/dL) or milli-international units per litre (mUI/L) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in fasting blood glucose level |
Measured in mg/dL or g/L or millimoles per litre (mmol/L) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in fasting total cholesterol |
Measured in mg/dl or g/L or mmol/L |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in fasting triglycerides |
Measured in mg/dL or g/L or mmol/L |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in fasting high-density lipoproteins (HDL) cholesterol |
Measured in mg/dL or g/L or mmol/L |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in fasting low-density lipoproteins (LDL) cholesterol |
Measured in mg/dL or g/L or mmol/L |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in fasting total cholesterol/HDL ratio |
Ratio of fasting total cholesterol and HDL |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in inhibin B |
Measured in picograms per milliliter (pg/mL) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in Anti-Müllerian Hormone (AMH) |
Measured in milligrams per milliliter (mg/mL) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in Luteinizing Hormone (LH) (female from 8 years old and male from 9 years old) |
Measured in mg/mL |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in Follicle-Stimulating Hormone (FSH) (female from 8 years old and male from 9 years old) |
Measured in International units per litre (UI/L) or mUI/mL |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in oestradiol (female from 8 years old and male from 9 years old) |
Measured in pg/mL |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in testosterone (female from 8 years old and male from 9 years old) |
Measured in ng/mL |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in dose of Norditropin® treatment |
Measured in milligram per kilogram per day (mg/kg/day) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Frequency of injections of Norditropin® treatment per week |
Count of events |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Number of modifications (change in dosage/ temporary stop) |
Count of events |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Reasons for treatment modifications |
Measured in number of participants under categorical variable - safety (occurrence of ARs or AEs), growth too fast, growth too slow, patient's or parents/LAR choice; and other |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Duration of temporary stop |
Measured in months |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Total duration of Norditropin® treatment |
Measured in years |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Total cumulative dose of Norditropin® treatment |
Measured in mg/kg |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Type and number of concomitant treatments |
Count of events |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Type and number of concomitant treatments linked to an adverse event |
Count of events |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in weight |
Measured in SDS |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in body mass index (BMI) |
Measured in kilogram per square metre (kg/m^2) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in bone age |
Measured in year |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in height velocity (cm/year) |
Measured in cm/year |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in height velocity (SDS/year) |
Measured in SDS/year |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Type and number of NS comorbidities |
Number of participants measured under categorical variables - Cardiac defects including pulmonary valve stenosis and hypertrophic cardiomyopathy, Orthopaedic issues (pectus, scoliosis) and Anorchia in males |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
| Secondary |
Change in Health-Related Quality of Life (HRQoL) score (PedsQL) |
PedsQL score used to assess the HRQoL, ranges from 1 to 100 (higher scores indicate better HRQoL) |
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) |
|
