Skip to main content
OpenTrials
Completed

NCT Number: NCT03435627

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Genetic Disorder Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Craniofacial Abnormalities Genetic Diseases, Inborn Heart Defects, Congenital Heart Diseases Musculoskeletal Abnormalities Musculoskeletal Diseases Noonan Syndrome Skin and Connective Tissue Diseases

Age range

3 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Novo Nordisk Investigational Site, Asahikawa, Hokkaido, Japan

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Somatropin

Drug

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

Primary outcomes

  1. Number of adverse drug reactions (ADR)

    Time frame: Weeks 0-208

    Count of events

Secondary outcomes

  1. Number of serious adverse drug reaction (SADR)

    Time frame: Week 0-208

    Count of events

  2. Number of serious adverse event (SAE)

    Time frame: Week 0-208

    Count of events

  3. Number of cardiac adverse event (AE)

    Time frame: Week 0-208

    Count of events

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

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

    Measured in ng/mL

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

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

    Measured in ng/mL

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

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

    Presented as standard deviation (SD) score

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

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

    Presented as SD score

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

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

    Measured in %

  9. Change in haemoglobin A1c (HbA1c) for naïve patients

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

    Measured in %

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

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

    Measured in IU/L

  11. Change in aspartate aminotransferase (AST) for naïve patients

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

    Measured in IU/L

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

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

    Measured in IU/L

  13. Change in alanine aminotransferase (ALT) for naïve patients

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

    Measured in IU/L

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

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

    Measured in mg/dL

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

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

    Measured in mg/dL

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

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

    Measured in mg/dL

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

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

    Measured in mg/dL

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

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

    Measured in mg/dL

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

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

    Measured in mg/dL

  20. Change in triglyceride (TG) for non-naïve patients

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

    Measured in mg/dL

  21. Change in triglyceride (TG) for naïve patients

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

    Measured in mg/dL

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

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

    Measured in μU/mL

  23. Change in thyroid stimulation hormone(TSH) for naïve patients

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

    Measured in μU/mL

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

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

    Measured in pg/mL

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

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

    Measured in pg/mL

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

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

    Measured in ng/mL

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

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

    Measured in ng/mL

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

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

    Measured in μ/L

  29. Change in white blood cell (WBC) for naïve patients

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

    Measured in μ/L

  30. Change in platelet (PLT) for non-naïve patients

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

    Measured in 10^4/μL

  31. Change in platelet (PLT) for naïve patients

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

    Measured in 10^4/μL

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

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

    Measured in year

  33. Change in bone age (BA) for naïve patients

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

    Measured in year

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

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

    Presented as ratio

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

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

    Presented as ratio

  36. Change in electrocardiogram (ECG) for non-naïve patients

    Time frame: 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.

  37. Change in electrocardiogram (ECG) for naïve patients

    Time frame: 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.

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

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

    Presented as SD score

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

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

    Presented as SD score

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

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

    Presented as SD score

  41. Height velocity standard deviation score (HVSDS) for naïve patients

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

    Presented as SD score

  42. Height velocity (HV) for non-naïve patients

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

    Measured in cm/year

  43. Height velocity (HV) for naïve patients

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

    Measured in cm/year

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Important dates

Study start
2018
Primary completion
2022
Study completion
2022
First posted
Feb 19, 2018
Registry last updated
Nov 14, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.