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

Novo Nordisk A/S71 enrolled

Overview

The purpose of this study is to collect information about safety and effectiveness for long term use of Norditropin®. Participants will attend the medical institution according to usual practice and receive medical care, as agreed with the study doctor.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Genetic Disorder Noonan Syndrome

Interventions

SomatropinDRUG

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

Eligibility

Sex: ALLMin age: 3 Years

Medical Language ↔ Plain English

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.

Locations (22)

Novo Nordisk Investigational Site

Asahikawa, Hokkaido, Japan

Outcomes

Primary Outcomes

Number of adverse drug reactions (ADR)

Count of events

Time frame: Weeks 0-208

Secondary Outcomes

Number of serious adverse drug reaction (SADR)

Count of events

Time frame: Week 0-208

Number of serious adverse event (SAE)

Count of events

Time frame: Week 0-208

Number of cardiac adverse event (AE)

Count of events

Time frame: Week 0-208

Change in insulin like growth factor I (IGF-I) for non-naïve patients

Measured in ng/mL

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 naïve patients

Measured in ng/mL

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

Presented as standard deviation (SD) score

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in insulin like growth factor I standard deviation score (IGF-I SDS) for naïve patients

Presented as SD score

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in haemoglobin A1c (HbA1c) for non-naïve patients

Data from ClinicalTrials.gov

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Novo Nordisk Investigational Site

Kanagawa, Japan

Novo Nordisk Investigational Site

Kanagawa, Japan

Novo Nordisk Investigational Site

Kyoto, Japan

Novo Nordisk Investigational Site

Kyoto, Japan

Novo Nordisk Investigational Site

Maebashi-shi, Gunma, Japan

Novo Nordisk Investigational Site

Miyazaki, Japan

Novo Nordisk Investigational Site

Nagoya, Aichi, Japan

Novo Nordisk Investigational Site

Niigata-shi, Niigata, Japan

...and 12 more locations

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in haemoglobin A1c (HbA1c) for naïve patients

Measured in %

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in aspartate aminotransferase (AST) for non-naïve patients

Measured in IU/L

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in aspartate aminotransferase (AST) for naïve patients

Measured in IU/L

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in alanine aminotransferase (ALT) for non-naïve patients

Measured in IU/L

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in alanine aminotransferase (ALT) for naïve patients

Measured in IU/L

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in total cholesterol (T-CHO) for non-naïve patients

Measured in mg/dL

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in total cholesterol (T-CHO) for naïve patients

Measured in mg/dL

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in high-density lipoprotein cholesterol (HDL-C) for non-naïve patients

Measured in mg/dL

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

Measured in mg/dL

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in Low-density lipoprotein cholesterol (LDL-C) for non-naïve patients

Measured in mg/dL

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

Measured in mg/dL

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in triglyceride (TG) for non-naïve patients

Measured in mg/dL

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in triglyceride (TG) for naïve patients

Measured in mg/dL

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in thyroid stimulation hormone(TSH) for non-naïve patients

Measured in μU/mL

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in thyroid stimulation hormone(TSH) for naïve patients

Measured in μU/mL

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in free triiodothyronine (FT-3) for non-naïve patients

Measured in pg/mL

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in free triiodothyronine (FT-3) for naïve patients

Measured in pg/mL

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in free thyroxine (FT-4) for non-naïve patients

Measured in ng/mL

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in free thyroxine (FT-4) for naïve patients

Measured in ng/mL

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in white blood cell (WBC) for non-naïve patients

Measured in μ/L

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in white blood cell (WBC) for naïve patients

Measured in μ/L

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in platelet (PLT) for non-naïve patients

Measured in 10\^4/μL

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in platelet (PLT) for naïve patients

Measured in 10\^4/μL

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in bone age (BA) for non-naïve patients

Measured in year

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in bone age (BA) for naïve patients

Measured in year

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in bone age/chronological age (BA/CA) for non-naïve patients

Presented as ratio

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in bone age/chronological age (BA/CA) for naïve patients

Presented as ratio

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in electrocardiogram (ECG) for non-naïve patients

Assessment of change in ECG is measured by categories recorded in case report forms (CRFs): normal, abnormal not clinically significant and abnormal clinically significant.

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in electrocardiogram (ECG) for naïve patients

Assessment of change in ECG is measured by categories recorded in CRFs: normal, abnormal not clinically significant and abnormal clinically significant.

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Change in height standard deviation score (HSDS) for non-naïve patients

Presented as SD score

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Change in height standard deviation score (HSDS) for naïve patients

Presented as SD score

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Height velocity standard deviation score (HVSDS) for non-naïve patients

Presented as SD score

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Height velocity standard deviation score (HVSDS) for naïve patients

Presented as SD score

Time frame: Weeks 0, 52, 104, 156, and 208 weeks

Height velocity (HV) for non-naïve patients

Measured in cm/year

Time frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks

Height velocity (HV) for naïve patients

Measured in cm/year

Time frame: Weeks 0, 52, 104, 156, and 208 weeks