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

Genetic Disorder Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03435627
• Other study ID #: GHLIQUID-4358
• Secondary ID: U1111-1191-3084
• Status: Completed
• Start date: February 26, 2018
• Est. completion date: January 18, 2022

Study information

• Verified date: November 2022
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 71
• Est. completion date: January 18, 2022
• Est. primary completion date: January 18, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Dwarfism
• Genetic Diseases, Inborn
• Genetic Disorder
• Noonan Syndrome
• Syndrome

Intervention

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

Locations

Country	Name	City	State
Japan	Novo Nordisk Investigational Site	Asahikawa, Hokkaido
Japan	Novo Nordisk Investigational Site	Fukuoka
Japan	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	Novo Nordisk Investigational Site	Oita
Japan	Novo Nordisk Investigational Site	Osaka
Japan	Novo Nordisk Investigational Site	Osaka
Japan	Novo Nordisk Investigational Site	Saitama-shi, Saitama
Japan	Novo Nordisk Investigational Site	Sapporo, Hokkaido
Japan	Novo Nordisk Investigational Site	Sendai-shi, Miyagi
Japan	Novo Nordisk Investigational Site	Shizuoka
Japan	Novo Nordisk Investigational Site	Tochigi
Japan	Novo Nordisk Investigational Site	Tokyo
Japan	Novo Nordisk Investigational Site	Tokyo
Japan	Novo Nordisk Investigational Site	Tokyo
Japan	Novo Nordisk Investigational Site	Tokyo

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of adverse drug reactions (ADR)	Count of events	Weeks 0-208
Secondary	Number of serious adverse drug reaction (SADR)	Count of events	Week 0-208
Secondary	Number of serious adverse event (SAE)	Count of events	Week 0-208
Secondary	Number of cardiac adverse event (AE)	Count of events	Week 0-208
Secondary	Change in insulin like growth factor I (IGF-I) for non-naïve patients	Measured in ng/mL	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in insulin like growth factor I (IGF-I) for naïve patients	Measured in ng/mL	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in insulin like growth factor I standard deviation score (IGF-I SDS) for non-naïve patients	Presented as standard deviation (SD) score	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in insulin like growth factor I standard deviation score (IGF-I SDS) for naïve patients	Presented as SD score	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in haemoglobin A1c (HbA1c) for non-naïve patients	Measured in %	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in haemoglobin A1c (HbA1c) for naïve patients	Measured in %	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in aspartate aminotransferase (AST) for non-naïve patients	Measured in IU/L	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in aspartate aminotransferase (AST) for naïve patients	Measured in IU/L	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in alanine aminotransferase (ALT) for non-naïve patients	Measured in IU/L	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in alanine aminotransferase (ALT) for naïve patients	Measured in IU/L	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in total cholesterol (T-CHO) for non-naïve patients	Measured in mg/dL	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in total cholesterol (T-CHO) for naïve patients	Measured in mg/dL	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in high-density lipoprotein cholesterol (HDL-C) for non-naïve patients	Measured in mg/dL	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in high-density lipoprotein cholesterol (HDL-C) for naïve patients	Measured in mg/dL	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in Low-density lipoprotein cholesterol (LDL-C) for non-naïve patients	Measured in mg/dL	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in Low-density lipoprotein cholesterol (LDL-C) for naïve patients	Measured in mg/dL	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in triglyceride (TG) for non-naïve patients	Measured in mg/dL	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in triglyceride (TG) for naïve patients	Measured in mg/dL	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in thyroid stimulation hormone(TSH) for non-naïve patients	Measured in µU/mL	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in thyroid stimulation hormone(TSH) for naïve patients	Measured in µU/mL	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in free triiodothyronine (FT-3) for non-naïve patients	Measured in pg/mL	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in free triiodothyronine (FT-3) for naïve patients	Measured in pg/mL	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in free thyroxine (FT-4) for non-naïve patients	Measured in ng/mL	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in free thyroxine (FT-4) for naïve patients	Measured in ng/mL	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in white blood cell (WBC) for non-naïve patients	Measured in µ/L	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in white blood cell (WBC) for naïve patients	Measured in µ/L	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in platelet (PLT) for non-naïve patients	Measured in 10^4/µL	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in platelet (PLT) for naïve patients	Measured in 10^4/µL	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in bone age (BA) for non-naïve patients	Measured in year	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in bone age (BA) for naïve patients	Measured in year	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in bone age/chronological age (BA/CA) for non-naïve patients	Presented as ratio	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in bone age/chronological age (BA/CA) for naïve patients	Presented as ratio	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	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.	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	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.	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Change in height standard deviation score (HSDS) for non-naïve patients	Presented as SD score	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Change in height standard deviation score (HSDS) for naïve patients	Presented as SD score	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Height velocity standard deviation score (HVSDS) for non-naïve patients	Presented as SD score	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Height velocity standard deviation score (HVSDS) for naïve patients	Presented as SD score	Weeks 0, 52, 104, 156, and 208 weeks
Secondary	Height velocity (HV) for non-naïve patients	Measured in cm/year	Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Secondary	Height velocity (HV) for naïve patients	Measured in cm/year	Weeks 0, 52, 104, 156, and 208 weeks