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

September 24, 2025 updated by: Novo Nordisk A/S
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 Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Enrolling by invitation

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
221

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Angers, France, 49033
        • Centre Hospitalier Universitaire D'Angers-2
      • Le Kremlin-Bicêtre, France, 94275
        • Ap-Hp-Hopital de Bicetre-2
      • Toulouse, France, 31059
        • Hopital Des Enfants-2

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Other

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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:
  • Somatropin
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:
  • Somatropin
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:
  • Somatropin

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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 (%)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
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)
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)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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 %
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
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)
Time Frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Measured in mg/mL
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)
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
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)
Time Frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Measured in pg/mL
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)
Time Frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Measured in ng/mL
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
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
Time Frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Count of events
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Change in weight
Time Frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Measured in SDS
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
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
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)
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)
From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novo Nordisk A/S

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Clinical Transparency dept. 2834, Novo Nordisk A/S

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 16, 2022

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 17, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 17, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 14, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 24, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 4, 2022

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 25, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 24, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GH-4831
  • U1111-1264-1805 (Other Identifier: World Health Organization (WHO))

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Drug and device information, study documents

Studies a U.S. FDA-regulated device product

No

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