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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1191-3084 | Other Identifier | World Health Organization (WHO) |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Norditropin® (naïve participants) | The treatment period of Norditropin® for naïve participants will be up to 208 weeks. |
| |
| Norditropin® (non-naïve participants) | The treatment period of Norditropin® for non-naïve participants will be up to 442 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Somatropin | Drug | Participants will be treated with commercially available Norditropin® (somatropin) according to routine clinical practice at the discretion of the treating physician. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of adverse drug reactions (ADR) | Count of events | Weeks 0-208 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of serious adverse drug reaction (SADR) | Count of events | Week 0-208 |
| Number of serious adverse event (SAE) | Count of events | Week 0-208 |
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Inclusion Criteria:
Exclusion Criteria:
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A total of 60 patients (consisting of non-naïve patients who were previously enrolled in study: GHLIQUID-4020 and naïve patients enrolled after approval of the Noonan indication in Japan) are planned to be included in this study.
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Reporting Anchor and Disclosure (1452) | Novo Nordisk A/S | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novo Nordisk Investigational Site | Asahikawa, Hokkaido | 078-8510 | Japan | |||
| Novo Nordisk Investigational Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41285480 | Derived | Muroya K, Kawai M, Yamagishi H, Endo T, Pietropoli A, Ferran JM, Horikawa R. Long-term effectiveness and safety of daily growth hormone therapy in Japanese children with Noonan syndrome: a post-marketing surveillance study. Endocr J. 2026 Feb 2;73(2):229-241. doi: 10.1507/endocrj.EJ25-0116. Epub 2025 Nov 22. |
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According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
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| Number of cardiac adverse event (AE) | Count of events | Week 0-208 |
| 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 |
| Change in insulin like growth factor I (IGF-I) for naïve patients | Measured in ng/mL | 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 | 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 | Weeks 0, 52, 104, 156, and 208 weeks |
| Change in haemoglobin A1c (HbA1c) for non-naïve patients | Measured in % | Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks |
| Change in haemoglobin A1c (HbA1c) for naïve patients | Measured in % | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in aspartate aminotransferase (AST) for naïve patients | Measured in IU/L | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in alanine aminotransferase (ALT) for naïve patients | Measured in IU/L | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in total cholesterol (T-CHO) for naïve patients | Measured in mg/dL | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in high-density lipoprotein cholesterol (HDL-C) for naïve patients | Measured in mg/dL | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in Low-density lipoprotein cholesterol (LDL-C) for naïve patients | Measured in mg/dL | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in triglyceride (TG) for naïve patients | Measured in mg/dL | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in thyroid stimulation hormone(TSH) for naïve patients | Measured in μU/mL | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in free triiodothyronine (FT-3) for naïve patients | Measured in pg/mL | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in free thyroxine (FT-4) for naïve patients | Measured in ng/mL | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in white blood cell (WBC) for naïve patients | Measured in μ/L | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in platelet (PLT) for naïve patients | Measured in 10^4/μL | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in bone age (BA) for naïve patients | Measured in year | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in bone age/chronological age (BA/CA) for naïve patients | Presented as ratio | 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. | 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. | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Change in height standard deviation score (HSDS) for naïve patients | Presented as SD score | Weeks 0, 52, 104, 156, and 208 weeks |
| 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 |
| Height velocity standard deviation score (HVSDS) for naïve patients | Presented as SD score | Weeks 0, 52, 104, 156, and 208 weeks |
| Height velocity (HV) for non-naïve patients | Measured in cm/year | Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks |
| Height velocity (HV) for naïve patients | Measured in cm/year | Weeks 0, 52, 104, 156, and 208 weeks |
| Fukuoka |
| 830-0011 |
| Japan |
| Novo Nordisk Investigational Site | Kanagawa | 216-8511 | Japan |
| Novo Nordisk Investigational Site | Kanagawa | 232-8555 | Japan |
| Novo Nordisk Investigational Site | Kyoto | 602-8566 | Japan |
| Novo Nordisk Investigational Site | Kyoto | 629-2261 | Japan |
| Novo Nordisk Investigational Site | Maebashi-shi, Gunma | 371-8511 | Japan |
| Novo Nordisk Investigational Site | Miyazaki | 889-1692 | Japan |
| Novo Nordisk Investigational Site | Nagoya, Aichi | 467-8602 | Japan |
| Novo Nordisk Investigational Site | Niigata-shi, Niigata | 951 8520 | Japan |
| Novo Nordisk Investigational Site | Osaka | 534-0021 | Japan |
| Novo Nordisk Investigational Site | Osaka | 594-1101 | Japan |
| Novo Nordisk Investigational Site | Ōita | 879-5593 | Japan |
| Novo Nordisk Investigational Site | Saitama-shi, Saitama | 330-8777 | Japan |
| Novo Nordisk Investigational Site | Sapporo, Hokkaido | 065-8611 | Japan |
| Novo Nordisk Investigational Site | Sendai-shi, Miyagi | 980 8574 | Japan |
| Novo Nordisk Investigational Site | Shizuoka | 431-3192 | Japan |
| Novo Nordisk Investigational Site | Tochigi | 329-0498 | Japan |
| Novo Nordisk Investigational Site | Tokyo | 157 8535 | Japan |
| Novo Nordisk Investigational Site | Tokyo | 160-8582 | Japan |
| Novo Nordisk Investigational Site | Tokyo | 162-8666 | Japan |
| Novo Nordisk Investigational Site | Tokyo | 183-8561 | Japan |
| ID | Term |
|---|---|
| D030342 | Genetic Diseases, Inborn |
| D009634 | Noonan Syndrome |
| ID | Term |
|---|---|
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D019465 | Craniofacial Abnormalities |
| D009139 | Musculoskeletal Abnormalities |
| D009140 | Musculoskeletal Diseases |
| D006330 | Heart Defects, Congenital |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D006331 | Heart Diseases |
| D000013 | Congenital Abnormalities |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
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| ID | Term |
|---|---|
| D019382 | Human Growth Hormone |
| ID | Term |
|---|---|
| D013006 | Growth Hormone |
| D010908 | Pituitary Hormones, Anterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
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