French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Norditropin.
- Who it may be relevant to
- Registry conditions: Noonan Syndrome. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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.
Interventions
- Drug Norditropin
Patients will be treated with commercially available Norditropin® according to routine clinical practice at the discretion of the treating physician.
Primary outcome measures
- Change in height standard deviation score [Time frame: From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)]
Secondary outcome measures (12)
- Number of adverse drug reactions (ADR) [Time frame: 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)]
- Number of serious adverse events (SAE) [Time frame: 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)]
- Education [Time frame: 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)]
- Educational measures [Time frame: 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)]
- Change in pulse rate [Time frame: 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)]
- Change in systolic blood pressure [Time frame: 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)]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
France · 3 centers
- Centre Hospitalier Universitaire D'Angers-2 — Angers
- Ap-Hp-Hopital de Bicetre-2 — Le Kremlin-Bicêtre
- Hopital Des Enfants-2 — Toulouse
Identifiers
NCT: NCT05308927 · GH-4831 · U1111-1264-1805