Clinical trial NCT05308927: French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome
Official title: French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Noonan Syndrome
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2022-04-04
Enrollment: 221
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The registry will include children treated with Norditropin® for short stature due to NS.

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.


Eligibility 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

Primary outcomes:
measure: Change in height standard deviation score
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Secondary outcomes:
measure: Number of adverse drug reactions (ADR)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Number of serious adverse drug reactions (SADR)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Number of serious adverse events (SAE)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Number of adverse events (AE)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Education
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: School level
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Educational measures
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Tanner stage
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in pulse rate
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in diastolic blood pressure
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in systolic blood pressure
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in corrected QT-interval (Electrocardiogram)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in echocardiogram parameters (mm)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in echocardiogram parameters (%)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in echocardiogram parameters (cm/s)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in complete blood count (CBC) (g/dL)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in CBC (G/L)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in Insulin-like growth factor 1 (IGF-1) level (ng/ml)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in IGF-1 level (SDS)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in Haemoglobin A1c (HbA1c) level
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in fasting insulin level
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in fasting blood glucose level
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in fasting total cholesterol
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in fasting triglycerides
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in fasting high-density lipoproteins (HDL) cholesterol
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in fasting low-density lipoproteins (LDL) cholesterol
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in fasting total cholesterol/HDL ratio
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in inhibin B
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in Anti-Müllerian Hormone (AMH)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in Luteinizing Hormone (LH) (female from 8 years old and male from 9 years old)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in Follicle-Stimulating Hormone (FSH) (female from 8 years old and male from 9 years old)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in oestradiol (female from 8 years old and male from 9 years old)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in testosterone (female from 8 years old and male from 9 years old)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in dose of Norditropin® treatment
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Frequency of injections of Norditropin® treatment per week
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Number of modifications (change in dosage/ temporary stop)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Reasons for treatment modifications
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Duration of temporary stop
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Total duration of Norditropin® treatment
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Total cumulative dose of Norditropin® treatment
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Type and number of concomitant treatments
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Type and number of concomitant treatments linked to an adverse event
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in weight
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in body mass index (BMI)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in bone age
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in height velocity (cm/year)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in height velocity (SDS/year)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Type and number of NS comorbidities
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
measure: Change in Health-Related Quality of Life (HRQoL) score (PedsQL)
timeFrame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)
Other outcomes:
Locations:
city: ANGERS cedex 09
country: France
facility: Novo Nordisk Investigational Site
city: Le Kremlin-bicetre
country: France
facility: Novo Nordisk Investigational Site
city: Toulouse cedex 9
country: France
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Transparency dept. 2834
role: STUDY_DIRECTOR
References: