This is a non-interventional registry of children treated with Norditropin® for short stature due to Noonan Syndrome (NS). This study aims to provide data on long-term growth evolution and safety of Norditropin® as well as Health Related Quality of Life (HRQoL) data. This registry will include the entirety of children treated with Norditropin® for short stature due to NS over the inclusion period. The decision to initiate treatment with commercially available Norditropin® is made by the patient/parents/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study.
Study Type
OBSERVATIONAL
Enrollment
221
Patients will be treated with commercially available Norditropin® according to routine clinical practice at the discretion of the treating physician.
Centre Hospitalier Universitaire D'Angers-2
Angers, France
Ap-Hp-Hopital de Bicetre-2
Le Kremlin-Bicêtre, France
Hopital Des Enfants-2
Toulouse, France
Change in height standard deviation score
Measured in standard deviation scores (SDS) ranging from -10 to +10
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Number of adverse drug reactions (ADR)
Count of events
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Number of serious adverse drug reactions (SADR)
Count of events
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Number of serious adverse events (SAE)
Count of events
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Number of adverse events (AE)
Count of events
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Education
Number of participants is measured under the categorical variables - standard education, education with assistance and educational measures
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
School level
Number of participants is measured under the variables - preschool, elementary school, secondary school and high school
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Educational measures
Number of participants is measured under the categorical variables - physiotherapy, psychomotricity, occupational therapy and speech therapy
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in pulse rate
Measured in beats per minute
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in diastolic blood pressure
Measured in millimeters of Mercury (mm Hg)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in systolic blood pressure
Measured in mm Hg
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in corrected QT-interval (Electrocardiogram)
Measured in millisecond (msec)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in echocardiogram parameters (%)
Change in Left Ventricular Fractional Shortening (LVFS) and Left Ventricular Ejection Fraction (LVEF) is measured in percentage (%)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in echocardiogram parameters (cm/s)
Change in Diastolic function (Ea and Aa) measured in centimeters per second (cm/s)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in complete blood count (CBC) (g/dL)
Change in Haemoglobin (Hb) measured in grams per deciLiter (g/dL)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in CBC (G/L)
Change in white blood cells, lymphocytes, monocytes and platelets measured in grams per liter (G/L)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in Insulin-like growth factor 1 (IGF-1) level (ng/ml)
Measured in nanograms per milliliter (ng/ml)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in IGF-1 level (SDS)
Measured in SDS
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in Haemoglobin A1c (HbA1c) level
Measured in %
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in fasting insulin level
Measured in milligrams per deciliter (mg/dL) or milli-international units per litre (mUI/L)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in fasting blood glucose level
Measured in mg/dL or g/L or millimoles per litre (mmol/L)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in fasting total cholesterol
Measured in mg/dl or g/L or mmol/L
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in fasting triglycerides
Measured in mg/dL or g/L or mmol/L
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in fasting high-density lipoproteins (HDL) cholesterol
Measured in mg/dL or g/L or mmol/L
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in fasting low-density lipoproteins (LDL) cholesterol
Measured in mg/dL or g/L or mmol/L
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in fasting total cholesterol/HDL ratio
Ratio of fasting total cholesterol and HDL
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in inhibin B
Measured in picograms per milliliter (pg/mL)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in Anti-Müllerian Hormone (AMH)
Measured in milligrams per milliliter (mg/mL)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in Luteinizing Hormone (LH) (female from 8 years old and male from 9 years old)
Measured in mg/mL
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in oestradiol (female from 8 years old and male from 9 years old)
Measured in pg/mL
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in testosterone (female from 8 years old and male from 9 years old)
Measured in ng/mL
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in dose of Norditropin® treatment
Measured in milligram per kilogram per day (mg/kg/day)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Frequency of injections of Norditropin® treatment per week
Count of events
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Number of modifications (change in dosage/ temporary stop)
Count of events
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Duration of temporary stop
Measured in months
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Total duration of Norditropin® treatment
Measured in years
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Total cumulative dose of Norditropin® treatment
Measured in mg/kg
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Type and number of concomitant treatments
Count of events
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Type and number of concomitant treatments linked to an adverse event
Count of events
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in weight
Measured in SDS
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in body mass index (BMI)
Measured in kilogram per square metre (kg/m\^2)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in bone age
Measured in year
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in height velocity (cm/year)
Measured in cm/year
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in height velocity (SDS/year)
Measured in SDS/year
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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