Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)

• Register: ClinicalTrials.gov
• Last refreshed on: 21 November 2022
• Main ID: NCT03435627
• Date of registration: 31/01/2018
• Prospective Registration: Yes
• Primary sponsor: Novo Nordisk A/S
• Public title: Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
• Scientific title: Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
• Date of first enrolment: February 26, 2018
• Target sample size: 71
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03435627
• Study type: Observational

Countries of recruitment

Japan

Contacts

• Name: Clinical Reporting Anchor and Disclosure (1452)
• Affiliation: Novo Nordisk A/S

Key inclusion & exclusion criteria

Inclusion Criteria:

- Signed informed consent obtained before any study-related activities (study-related
activities are any procedure related to recording of data according to the protocol).

- The decision to initiate treatment with commercially available Norditropin® has been
made by the patient/Legally Acceptable Representative (LAR) and the treating physician
before and independently from the decision to include the patient in this study.

- For non-naïve patients; patients who were previously enrolled in study: GHLIQUID-4020.

- For naïve patients; short stature due to Noonan syndrome diagnosed by the physician
and a decision to initiate treatment with Norditropin® has been made by the
patient/parent and the physician. At study sites, all patients will be registered
consecutively from the first patient after approval date (consecutively registered
system).

- Male or female, 3 years old or over, bone age: less than 17 years old for male / less
than 15 years old for female.

Exclusion Criteria:

- Previous participation in this study. Participation is defined as having given
informed consent in this study.

- Known or suspected allergy to study products or related products.

- In case of naïve patients, patients who have received growth hormone (GH) products for
treatment of indication other than short stature due to Noonan syndrome before
approval date of Noonan indication.



Age minimum: 3 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Genetic Disorder

Noonan Syndrome

Intervention(s)

Drug: Somatropin

Primary Outcome(s)

Number of adverse drug reactions (ADR) [Time Frame: Weeks 0-208]

Secondary Outcome(s)

Change in alanine aminotransferase (ALT) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in bone age/chronological age (BA/CA) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in alanine aminotransferase (ALT) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in free triiodothyronine (FT-3) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in insulin like growth factor I standard deviation score (IGF-I SDS) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in total cholesterol (T-CHO) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in triglyceride (TG) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in white blood cell (WBC) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Height velocity (HV) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in aspartate aminotransferase (AST) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in aspartate aminotransferase (AST) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in platelet (PLT) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in total cholesterol (T-CHO) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in free thyroxine (FT-4) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in insulin like growth factor I (IGF-I) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in bone age (BA) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in height standard deviation score (HSDS) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Height velocity (HV) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in free triiodothyronine (FT-3) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in haemoglobin A1c (HbA1c) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in height standard deviation score (HSDS) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in bone age (BA) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Number of cardiac adverse event (AE) [Time Frame: Week 0-208]

Change in bone age/chronological age (BA/CA) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in free thyroxine (FT-4) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in Low-density lipoprotein cholesterol (LDL-C) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Height velocity standard deviation score (HVSDS) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Number of serious adverse event (SAE) [Time Frame: Week 0-208]

Change in electrocardiogram (ECG) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in Low-density lipoprotein cholesterol (LDL-C) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in insulin like growth factor I (IGF-I) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in thyroid stimulation hormone(TSH) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in platelet (PLT) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in insulin like growth factor I standard deviation score (IGF-I SDS) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in high-density lipoprotein cholesterol (HDL-C) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in white blood cell (WBC) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Height velocity standard deviation score (HVSDS) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in thyroid stimulation hormone(TSH) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in electrocardiogram (ECG) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in haemoglobin A1c (HbA1c) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Change in high-density lipoprotein cholesterol (HDL-C) for naïve patients [Time Frame: Weeks 0, 52, 104, 156, and 208 weeks]

Change in triglyceride (TG) for non-naïve patients [Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks]

Number of serious adverse drug reaction (SADR) [Time Frame: Week 0-208]

Secondary ID(s)

U1111-1191-3084

GHLIQUID-4358

Source(s) of Monetary Support

Please refer to primary and secondary sponsors