Clinical trial

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

Name

GH-4831

Description

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.

Trial arms

Trial start

2022-03-16

Estimated PCD

2028-03-17

Trial end

2028-03-17

Treatment

Norditropin

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

Arms:

Incident patients, Prevalent patients - finished growth upon inclusion, Prevalent patients - not finished growth upon inclusion

Other names:

Somatropin

Size

221

Primary endpoint

Change in height standard deviation score

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

Protocol

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'OTHER'}, 'enrollmentInfo': {'count': 221, 'type': 'ESTIMATED'}}

Updated at

2023-05-22

1 organization

1 product

1 indication

Product

Norditropin

Indication

Noonan syndrome

Organization

Novo Nordisk