CClinicalTrials.gg
Sign In
CompletedNCT03435627Updated Nov 14, 2022

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

An observational study in Genetic Disorder and Noonan Syndrome, sponsored by Novo Nordisk A/S. Completed at 22 sites in Japan. Open to participants aged 3 Years and older. Per ClinicalTrials.gov, last updated 2022-11-14.

Sponsored by Novo Nordisk A/S · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
71
Ages
3 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • Genetic Disorder
  • Noonan Syndrome
03

Who can participate

Ages eligible
3 Years and older
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

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.

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

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.
04

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
71 participants (actual)
Patient registry
No

Groups and cohorts

  • Norditropin® (naïve participants)

    The treatment period of Norditropin® for naïve participants will be up to 208 weeks.

    Drug: Somatropin

  • Norditropin® (non-naïve participants)

    The treatment period of Norditropin® for non-naïve participants will be up to 442 weeks.

    Drug: Somatropin

Interventions

  • DrugSomatropin

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

05

What researchers measure

Primary outcomes

  1. Number of adverse drug reactions (ADR)

    Count of events

    Time frame: Weeks 0-208

Secondary outcomes

  1. Number of serious adverse drug reaction (SADR)

    Count of events

    Time frame: Week 0-208

  2. Number of serious adverse event (SAE)

    Count of events

    Time frame: Week 0-208

  3. Number of cardiac adverse event (AE)

    Count of events

    Time frame: Week 0-208

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

    Measured in ng/mL

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

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

    Measured in ng/mL

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

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

    Presented as standard deviation (SD) score

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

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

    Presented as SD score

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

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

    Measured in %

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

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

    Measured in %

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

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

    Measured in IU/L

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

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

    Measured in IU/L

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

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

    Measured in IU/L

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

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

    Measured in IU/L

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

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

    Measured in mg/dL

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

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

    Measured in mg/dL

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

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

    Measured in mg/dL

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

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

    Measured in mg/dL

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

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

    Measured in mg/dL

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

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

    Measured in mg/dL

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

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

    Measured in mg/dL

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

  21. Change in triglyceride (TG) for naïve patients

    Measured in mg/dL

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

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

    Measured in μU/mL

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

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

    Measured in μU/mL

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

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

    Measured in pg/mL

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

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

    Measured in pg/mL

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

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

    Measured in ng/mL

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

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

    Measured in ng/mL

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

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

    Measured in μ/L

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

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

    Measured in μ/L

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

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

    Measured in 10\^4/μL

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

  31. Change in platelet (PLT) for naïve patients

    Measured in 10\^4/μL

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

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

    Measured in year

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

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

    Measured in year

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

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

    Presented as ratio

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

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

    Presented as ratio

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

  36. 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.

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

  37. 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.

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

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

    Presented as SD score

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

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

    Presented as SD score

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

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

    Presented as SD score

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

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

    Presented as SD score

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

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

    Measured in cm/year

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

  43. Height velocity (HV) for naïve patients

    Measured in cm/year

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

06

Study locations

22 sites
  • Novo Nordisk Investigational Site
    Asahikawa, Hokkaido, 078-8510, Japan
  • Novo Nordisk Investigational Site
    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
    Oita, 879-5593, Japan
  • Novo Nordisk Investigational Site
    Osaka, 534-0021, Japan
  • Novo Nordisk Investigational Site
    Osaka, 594-1101, 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
07

References and documents

Individual participant data

Plan to share: Yes — According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT03435627
Lead sponsor
Novo Nordisk A/S
Responsible party
Sponsor
First posted
Feb 19, 2018
Start date
Feb 26, 2018
Primary completion
Jan 18, 2022
Completion
Jan 18, 2022
Last update
Nov 14, 2022

Study contacts

Clinical Reporting Anchor and Disclosure (1452)
study director · Novo Nordisk A/S

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Nov 2022. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Dwarfism→

Other studies from this sponsor

Novo Nordisk A/S

Start the discussion