Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
- Registration Number
- NCT03435627
- Lead Sponsor
- Novo Nordisk A/S
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 71
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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Norditropin® (naïve participants) Somatropin The treatment period of Norditropin® for naïve participants will be up to 208 weeks. Norditropin® (non-naïve participants) Somatropin The treatment period of Norditropin® for non-naïve participants will be up to 442 weeks.
- Primary Outcome Measures
Name Time Method Number of adverse drug reactions (ADR) Weeks 0-208 Count of events
- Secondary Outcome Measures
Name Time Method Change in insulin like growth factor I standard deviation score (IGF-I SDS) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Presented as SD score
Change in high-density lipoprotein cholesterol (HDL-C) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in mg/dL
Change in Low-density lipoprotein cholesterol (LDL-C) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in mg/dL
Number of serious adverse event (SAE) Week 0-208 Count of events
Number of cardiac adverse event (AE) Week 0-208 Count of events
Change in insulin like growth factor I (IGF-I) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in ng/mL
Change in insulin like growth factor I standard deviation score (IGF-I SDS) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Presented as standard deviation (SD) score
Change in aspartate aminotransferase (AST) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in IU/L
Change in alanine aminotransferase (ALT) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in IU/L
Change in total cholesterol (T-CHO) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in mg/dL
Change in high-density lipoprotein cholesterol (HDL-C) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in mg/dL
Change in platelet (PLT) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in 10\^4/μL
Change in bone age (BA) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in year
Change in bone age (BA) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in year
Change in bone age/chronological age (BA/CA) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Presented as ratio
Change in electrocardiogram (ECG) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Assessment of change in ECG is measured by categories recorded in case report forms (CRFs): normal, abnormal not clinically significant and abnormal clinically significant.
Change in electrocardiogram (ECG) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Assessment of change in ECG is measured by categories recorded in CRFs: normal, abnormal not clinically significant and abnormal clinically significant.
Change in height standard deviation score (HSDS) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Presented as SD score
Change in insulin like growth factor I (IGF-I) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in ng/mL
Number of serious adverse drug reaction (SADR) Week 0-208 Count of events
Change in aspartate aminotransferase (AST) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in IU/L
Height velocity standard deviation score (HVSDS) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Presented as SD score
Change in haemoglobin A1c (HbA1c) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in %
Change in total cholesterol (T-CHO) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in mg/dL
Change in triglyceride (TG) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in mg/dL
Change in triglyceride (TG) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in mg/dL
Change in free triiodothyronine (FT-3) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in pg/mL
Change in free thyroxine (FT-4) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in ng/mL
Change in white blood cell (WBC) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in μ/L
Change in platelet (PLT) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in 10\^4/μL
Change in bone age/chronological age (BA/CA) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Presented as ratio
Change in haemoglobin A1c (HbA1c) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in %
Change in alanine aminotransferase (ALT) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in IU/L
Change in Low-density lipoprotein cholesterol (LDL-C) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in mg/dL
Change in thyroid stimulation hormone(TSH) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in μU/mL
Change in white blood cell (WBC) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in μ/L
Change in thyroid stimulation hormone(TSH) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in μU/mL
Change in free triiodothyronine (FT-3) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in pg/mL
Change in free thyroxine (FT-4) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in ng/mL
Change in height standard deviation score (HSDS) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Presented as SD score
Height velocity (HV) for non-naïve patients Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks Measured in cm/year
Height velocity (HV) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Measured in cm/year
Height velocity standard deviation score (HVSDS) for naïve patients Weeks 0, 52, 104, 156, and 208 weeks Presented as SD score
Trial Locations
- Locations (1)
Novo Nordisk Investigational Site
🇯🇵Tokyo, Japan