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NCT Number: NCT05308927

French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome

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.

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Key information

Conditions

Noonan Syndrome Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Craniofacial Abnormalities Heart Defects, Congenital Heart Diseases Musculoskeletal Abnormalities Musculoskeletal Diseases Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Centre Hospitalier Universitaire D'Angers-2, Angers, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Norditropin

Drug

Patients will be treated with commercially available Norditropin® according to routine clinical practice at the discretion of the treating physician.

Other names: Somatropin

Primary outcomes

  1. Change in height standard deviation score

    Time frame: 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 outcomes

  1. Number of adverse drug reactions (ADR)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Count of events

  2. Number of serious adverse drug reactions (SADR)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Count of events

  3. Number of serious adverse events (SAE)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Count of events

  4. Number of adverse events (AE)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Count of events

  5. Education

    Time frame: 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

  6. School level

    Time frame: 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

  7. Educational measures

    Time frame: 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

  8. Tanner stage

    Time frame: 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

  9. Change in pulse rate

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in beats per minute

  10. Change in diastolic blood pressure

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in millimeters of Mercury (mm Hg)

  11. Change in systolic blood pressure

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in mm Hg

  12. Change in corrected QT-interval (Electrocardiogram)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in millisecond (msec)

  13. Change in echocardiogram parameters (mm)

    Time frame: 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)

  14. Change in echocardiogram parameters (%)

    Time frame: 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 (%)

  15. Change in echocardiogram parameters (cm/s)

    Time frame: 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)

  16. Change in complete blood count (CBC) (g/dL)

    Time frame: 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)

  17. Change in CBC (G/L)

    Time frame: 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)

  18. Change in Insulin-like growth factor 1 (IGF-1) level (ng/ml)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in nanograms per milliliter (ng/ml)

  19. Change in IGF-1 level (SDS)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in SDS

  20. Change in Haemoglobin A1c (HbA1c) level

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in %

  21. Change in fasting insulin level

    Time frame: 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)

  22. Change in fasting blood glucose level

    Time frame: 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)

  23. Change in fasting total cholesterol

    Time frame: 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

  24. Change in fasting triglycerides

    Time frame: 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

  25. Change in fasting high-density lipoproteins (HDL) cholesterol

    Time frame: 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

  26. Change in fasting low-density lipoproteins (LDL) cholesterol

    Time frame: 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

  27. Change in fasting total cholesterol/HDL ratio

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Ratio of fasting total cholesterol and HDL

  28. Change in inhibin B

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in picograms per milliliter (pg/mL)

  29. Change in Anti-Müllerian Hormone (AMH)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in milligrams per milliliter (mg/mL)

  30. Change in Luteinizing Hormone (LH) (female from 8 years old and male from 9 years old)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in mg/mL

  31. Change in Follicle-Stimulating Hormone (FSH) (female from 8 years old and male from 9 years old)

    Time frame: 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

  32. Change in oestradiol (female from 8 years old and male from 9 years old)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in pg/mL

  33. Change in testosterone (female from 8 years old and male from 9 years old)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in ng/mL

  34. Change in dose of Norditropin® treatment

    Time frame: 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)

  35. Frequency of injections of Norditropin® treatment per week

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Count of events

  36. Number of modifications (change in dosage/ temporary stop)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Count of events

  37. Reasons for treatment modifications

    Time frame: 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

  38. Duration of temporary stop

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in months

  39. Total duration of Norditropin® treatment

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in years

  40. Total cumulative dose of Norditropin® treatment

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in mg/kg

  41. Type and number of concomitant treatments

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Count of events

  42. Type and number of concomitant treatments linked to an adverse event

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Count of events

  43. Change in weight

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in SDS

  44. Change in body mass index (BMI)

    Time frame: 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)

  45. Change in bone age

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in year

  46. Change in height velocity (cm/year)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in cm/year

  47. Change in height velocity (SDS/year)

    Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

    Measured in SDS/year

  48. Type and number of NS comorbidities

    Time frame: 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

  49. Change in Health-Related Quality of Life (HRQoL) score (PedsQL)

    Time frame: 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)

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Important dates

Study start
2022
Primary completion
2028
Study completion
2028
First posted
Apr 4, 2022
Registry last updated
Sep 25, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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