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

Novo Nordisk

Status: Invitation-only

Conditions: Noonan Syndrome

Treatments: Drug: Norditropin

Study type: Observational

Funder types: Industry

Identifiers

NCT05308927

U1111-1264-1805 (Other Identifier)

GH-4831

Details and patient eligibility

About

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.

Enrollment: 221 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

221 participants in 3 patient groups

Incident patients

Description:

Children initiated Norditropin® upon their inclusion in the study but independently from the decision to participate in this study

Treatment:

Drug: Norditropin

Prevalent patients - finished growth upon inclusion

Description:

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

Treatment:

Drug: Norditropin

Prevalent patients - not finished growth upon inclusion

Description:

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

Treatment:

Drug: Norditropin