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Noonan SyndromeNCT05308927

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

An observational study of Noonan Syndrome, run by Novo Nordisk A/S at 3 sites in France.

Enrolling by invitation

Enrolling by invitation. The study team selects and invites participants, usually from an existing group of patients.

Sites and contacts
Official record

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Trial 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. Read more about this study

Who is it for?
Any age
Conditions the study is for
Noonan Syndrome
What is being tested?
Norditropin
How many people
221 participants
Where
3 sitesFrance
Study dates
16 Mar 2022to 17 Mar 2028
Phase
Observational

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Study population: The registry will include children treated with Norditropin® for short stature due to NS.

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

3 sites in 1 country. Contact the study team or a site near you to ask about taking part.

France3 sites

  • Centre Hospitalier Universitaire D'Angers-2

    Angers, 49033

  • Ap-Hp-Hopital de Bicetre-2

    Le Kremlin-Bicêtre, 94275

  • Hopital Des Enfants-2

    Toulouse, 31059

Other trials in France

About this study

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.

Conditions
Noonan Syndrome
Being tested
Norditropin
Medical subject headings
Noonan Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NorditropinDrug

    Also called Somatropin

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

Study groups in NCT05308927
Study groupRoleWhat they receive
Incident patientsChildren initiated Norditropin® upon their inclusion in the study but independently from the decision to participate in this study
Receives: Drug: Norditropin
Prevalent patients - finished growth upon inclusionChildren were already treated with Norditropin® before their inclusion in the study and finished their growth upon their inclusion. Data collected retrospectively from medical records
Receives: Drug: Norditropin
Prevalent patients - not finished growth upon inclusionChildren 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
Receives: Drug: Norditropin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in height standard deviation score

    Measured in standard deviation scores (SDS) ranging from -10 to +10

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

Secondary outcomes (49)

  1. Number of adverse drug reactions (ADR)

    Count of events

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

  2. Number of serious adverse drug reactions (SADR)

    Count of events

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

  3. Number of serious adverse events (SAE)

    Count of events

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

  4. Number of adverse events (AE)

    Count of events

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

  5. Education

    Number of participants is measured under the categorical variables - standard education, education with assistance and educational measures

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

  6. School level

    Number of participants is measured under the variables - preschool, elementary school, secondary school and high school

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

  7. Educational measures

    Number of participants is measured under the categorical variables - physiotherapy, psychomotricity, occupational therapy and speech therapy

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

  8. 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

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

  9. Change in pulse rate

    Measured in beats per minute

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

  10. Change in diastolic blood pressure

    Measured in millimeters of Mercury (mm Hg)

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

  11. Change in systolic blood pressure

    Measured in mm Hg

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

  12. Change in corrected QT-interval (Electrocardiogram)

    Measured in millisecond (msec)

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

  13. 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)

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

  14. Change in echocardiogram parameters (%)

    Change in Left Ventricular Fractional Shortening (LVFS) and Left Ventricular Ejection Fraction (LVEF) is measured in percentage (%)

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

  15. Change in echocardiogram parameters (cm/s)

    Change in Diastolic function (Ea and Aa) measured in centimeters per second (cm/s)

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

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

    Change in Haemoglobin (Hb) measured in grams per deciLiter (g/dL)

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

  17. Change in CBC (G/L)

    Change in white blood cells, lymphocytes, monocytes and platelets measured in grams per liter (G/L)

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

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

    Measured in nanograms per milliliter (ng/ml)

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

  19. Change in IGF-1 level (SDS)

    Measured in SDS

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

  20. Change in Haemoglobin A1c (HbA1c) level

    Measured in %

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

  21. Change in fasting insulin level

    Measured in milligrams per deciliter (mg/dL) or milli-international units per litre (mUI/L)

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

  22. Change in fasting blood glucose level

    Measured in mg/dL or g/L or millimoles per litre (mmol/L)

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

  23. Change in fasting total cholesterol

    Measured in mg/dl or g/L or mmol/L

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

  24. Change in fasting triglycerides

    Measured in mg/dL or g/L or mmol/L

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

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

    Measured in mg/dL or g/L or mmol/L

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

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

    Measured in mg/dL or g/L or mmol/L

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

  27. Change in fasting total cholesterol/HDL ratio

    Ratio of fasting total cholesterol and HDL

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

  28. Change in inhibin B

    Measured in picograms per milliliter (pg/mL)

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

  29. Change in Anti-Müllerian Hormone (AMH)

    Measured in milligrams per milliliter (mg/mL)

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

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

    Measured in mg/mL

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

  31. 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

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

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

    Measured in pg/mL

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

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

    Measured in ng/mL

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

  34. Change in dose of Norditropin® treatment

    Measured in milligram per kilogram per day (mg/kg/day)

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

  35. Frequency of injections of Norditropin® treatment per week

    Count of events

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

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

    Count of events

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

  37. 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

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

  38. Duration of temporary stop

    Measured in months

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

  39. Total duration of Norditropin® treatment

    Measured in years

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

  40. Total cumulative dose of Norditropin® treatment

    Measured in mg/kg

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

  41. Type and number of concomitant treatments

    Count of events

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

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

    Count of events

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

  43. Change in weight

    Measured in SDS

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

  44. Change in body mass index (BMI)

    Measured in kilogram per square metre (kg/m^2)

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

  45. Change in bone age

    Measured in year

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

  46. Change in height velocity (cm/year)

    Measured in cm/year

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

  47. Change in height velocity (SDS/year)

    Measured in SDS/year

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

  48. 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

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

  49. 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)

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Apr 2022
  2. Study start16 Mar 2022
  3. Primary completion17 Mar 2028 (estimated)
  4. Study completion17 Mar 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Other
Enrolment
221 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05308927
Sponsor's protocol ID
GH-4831
Other IDs
U1111-1264-1805
Status last verified
Oct 2026
First submitted
14 Mar 2022
Last updated
8 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated device
No
Individual data sharing
Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

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