French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome
- Conditions
- Noonan Syndrome
- Interventions
- Registration Number
- NCT05308927
- Lead Sponsor
- Novo Nordisk A/S
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 221
- Patients with a clinical and/or genetic diagnosis of NS
- Patients who are treated with Norditropin® (already treated or initiating) and who are followed in a participating center
- The decision to initiate treatment with commercially available Norditropin® has been 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
- Patients/Parents/LAR opposed to the collection and processing of their children's medical data
- Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Incident patients Norditropin Children initiated Norditropin® upon their inclusion in the study but independently from the decision to participate in this study Prevalent patients - finished growth upon inclusion Norditropin Children were already treated with Norditropin® before their inclusion in the study and finished their growth upon their inclusion. Data collected retrospectively from medical records Prevalent patients - not finished growth upon inclusion Norditropin Children were already treated with Norditropin® before their inclusion in the study and did not finish their growth upon inclusion. Data collected both retrospectively and prospectively
- Primary Outcome Measures
Name Time Method Change in height standard deviation score From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in standard deviation scores (SDS) ranging from -10 to +10
- Secondary Outcome Measures
Name Time Method Change in diastolic blood pressure From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in millimeters of Mercury (mm Hg)
Number of adverse drug reactions (ADR) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Count of events
Number of serious adverse events (SAE) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Count of events
Change in Haemoglobin A1c (HbA1c) level From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in %
Change in fasting total cholesterol From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in mg/dl or g/L or mmol/L
Change in fasting triglycerides From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in mg/dL or g/L or mmol/L
Number of serious adverse drug reactions (SADR) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Count of events
Number of adverse events (AE) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Count of events
Education From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Number of participants is measured under the categorical variables - standard education, education with assistance and educational measures
School level From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Number of participants is measured under the variables - preschool, elementary school, secondary school and high school
Educational measures From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Number of participants is measured under the categorical variables - physiotherapy, psychomotricity, occupational therapy and speech therapy
Tanner stage From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) 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
Change in pulse rate From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in beats per minute
Change in systolic blood pressure From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in mm Hg
Change in echocardiogram parameters (cm/s) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Change in Diastolic function (Ea and Aa) measured in centimeters per second (cm/s)
Change in fasting low-density lipoproteins (LDL) cholesterol From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in mg/dL or g/L or mmol/L
Change in Follicle-Stimulating Hormone (FSH) (female from 8 years old and male from 9 years old) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in International units per litre (UI/L) or mUI/mL
Frequency of injections of Norditropin® treatment per week From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Count of events
Reasons for treatment modifications From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) 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
Type and number of concomitant treatments linked to an adverse event From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Count of events
Change in body mass index (BMI) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in kilogram per square metre (kg/m\^2)
Change in corrected QT-interval (Electrocardiogram) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in millisecond (msec)
Change in echocardiogram parameters (mm) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Change in Left ventricular interventricular septal maximum thickness in diastole and Left ventricular end diastolic diameter is measured in millimeter (mm)
Change in echocardiogram parameters (%) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Change in Left Ventricular Fractional Shortening (LVFS) and Left Ventricular Ejection Fraction (LVEF) is measured in percentage (%)
Change in complete blood count (CBC) (g/dL) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Change in Haemoglobin (Hb) measured in grams per deciLiter (g/dL)
Change in CBC (G/L) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Change in white blood cells, lymphocytes, monocytes and platelets measured in grams per liter (G/L)
Change in Insulin-like growth factor 1 (IGF-1) level (ng/ml) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in nanograms per milliliter (ng/ml)
Change in IGF-1 level (SDS) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in SDS
Change in fasting insulin level From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in milligrams per deciliter (mg/dL) or milli-international units per litre (mUI/L)
Change in fasting blood glucose level From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in mg/dL or g/L or millimoles per litre (mmol/L)
Change in fasting high-density lipoproteins (HDL) cholesterol From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in mg/dL or g/L or mmol/L
Change in Luteinizing Hormone (LH) (female from 8 years old and male from 9 years old) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in mg/mL
Change in oestradiol (female from 8 years old and male from 9 years old) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in pg/mL
Change in testosterone (female from 8 years old and male from 9 years old) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in ng/mL
Change in weight From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in SDS
Change in fasting total cholesterol/HDL ratio From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Ratio of fasting total cholesterol and HDL
Total duration of Norditropin® treatment From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in years
Total cumulative dose of Norditropin® treatment From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in mg/kg
Change in bone age From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in year
Change in height velocity (cm/year) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in cm/year
Change in height velocity (SDS/year) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in SDS/year
Change in inhibin B From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in picograms per milliliter (pg/mL)
Change in Anti-Müllerian Hormone (AMH) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in milligrams per milliliter (mg/mL)
Change in dose of Norditropin® treatment From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in milligram per kilogram per day (mg/kg/day)
Number of modifications (change in dosage/ temporary stop) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Count of events
Duration of temporary stop From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Measured in months
Type and number of concomitant treatments From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Count of events
Type and number of NS comorbidities From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) Number of participants measured under categorical variables - Cardiac defects including pulmonary valve stenosis and hypertrophic cardiomyopathy, Orthopaedic issues (pectus, scoliosis) and Anorchia in males
Change in Health-Related Quality of Life (HRQoL) score (PedsQL) From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years) PedsQL score used to assess the HRQoL, ranges from 1 to 100 (higher scores indicate better HRQoL)
Trial Locations
- Locations (1)
Novo Nordisk Investigational Site
🇫🇷Toulouse cedex 9, France