French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Incident patients Children initiated Norditropin® upon their inclusion in the study but independently from the decision to participate in this study |
Drug: Norditropin
Patients will be treated with commercially available Norditropin® according to routine clinical practice at the discretion of the treating physician.
Other Names:
|
| Prevalent patients - finished growth upon inclusion 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 |
Drug: Norditropin
Patients will be treated with commercially available Norditropin® according to routine clinical practice at the discretion of the treating physician.
Other Names:
|
| Prevalent patients - not finished growth upon inclusion 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 |
Drug: Norditropin
Patients will be treated with commercially available Norditropin® according to routine clinical practice at the discretion of the treating physician.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- 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
- 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 drug reactions (SADR) [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
- 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 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)
- 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 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 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 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 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 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 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
- 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 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 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
- 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 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 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
- 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 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)
- 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
- 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
- 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
- Duration of temporary stop [From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
Measured in months
- 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
- 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 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 weight [From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
Measured in SDS
- 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 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
- 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)
Eligibility Criteria
Criteria
Inclusion Criteria:
-
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
Exclusion Criteria:
-
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Novo Nordisk Investigational Site | Le Kremlin-bicetre | France | 94275 | |
| 2 | Novo Nordisk Investigational Site | Toulouse cedex 9 | France | 31059 |
Sponsors and Collaborators
- Novo Nordisk A/S
Investigators
- Study Director: Clinical Transparency dept. 2834, Novo Nordisk A/S
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- GH-4831
- U1111-1264-1805