Genetic Disorder, Noonan Syndrome
Conditions
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.
Interventions
DRUGSomatropin
Participants will be treated with commercially available Norditropin® (somatropin) according to routine clinical practice at the discretion of the treating physician.
Sponsors
Novo Nordisk A/S
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of adverse drug reactions (ADR) | Weeks 0-208 | Count of events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in platelet (PLT) for naïve patients | Weeks 0, 52, 104, 156, and 208 weeks | Measured in 10\^4/μL |
| Number of serious adverse drug reaction (SADR) | Week 0-208 | Count of events |
| 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 non-naïve patients | Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks | Measured in ng/mL |
| 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 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 haemoglobin A1c (HbA1c) for naïve patients | Weeks 0, 52, 104, 156, and 208 weeks | Measured in % |
| 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 |
| 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 non-naïve patients | Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 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 total cholesterol (T-CHO) for naïve patients | Weeks 0, 52, 104, 156, and 208 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 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 |
| 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 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 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 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 non-naïve patients | Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks | Measured in pg/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 non-naïve patients | Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks | Measured in ng/mL |
| Change in free thyroxine (FT-4) for naïve patients | Weeks 0, 52, 104, 156, and 208 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 white blood cell (WBC) for naïve patients | Weeks 0, 52, 104, 156, and 208 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 (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 non-naïve patients | Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks | Presented as ratio |
| 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 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 % |
| 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 |
| Height velocity standard deviation score (HVSDS) for naïve patients | Weeks 0, 52, 104, 156, and 208 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 |
| Change in height standard deviation score (HSDS) for naïve patients | Weeks 0, 52, 104, 156, and 208 weeks | Presented as SD score |
Countries
Japan
Outcome results
None listed