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

NCT05308927 | Enrolling By Invitation

• 2 other identifiers: GH-4831U1111-1264-1805
• Study Type: observational
• Target: 221
• Locations: 1 country
• Sites: 3

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.

Conditions

Noonan Syndrome

Outcome Measures

Primary Outcomes (1)

• Change in height standard deviation score

  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 Outcomes (49)

• Number of adverse drug reactions (ADR)

  From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

• Number of serious adverse drug reactions (SADR)

  From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

• Number of serious adverse events (SAE)

  From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

• Number of adverse events (AE)

  From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

• Education

  From start of Norditropin® treatment (day 0) until end of follow-up (up to 6 years)

Study Arms (3)

Incident patients

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

Drug: Norditropin

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

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

Interventions

Norditropin DRUG

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

Also known as: Somatropin

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

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (3)

Centre Hospitalier Universitaire D'Angers-2

Angers, 49033, France

Location

Ap-Hp-Hopital de Bicetre-2

Le Kremlin-Bicêtre, 94275, France

Location

Hopital Des Enfants-2

Toulouse, 31059, France

Location

MeSH Terms

Conditions

Noonan Syndrome

Interventions

Human Growth Hormone

Condition Hierarchy (Ancestors)

Craniofacial Abnormalities; Musculoskeletal Abnormalities; Musculoskeletal Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Clinical Transparency dept. 2834

  Novo Nordisk A/S

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 14, 2022
• First Posted: April 4, 2022
• Study Start: March 16, 2022
• Primary Completion (Estimated): March 17, 2028
• Study Completion (Estimated): March 17, 2028
• Last Updated: September 25, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

FR (3)