Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03435627
Recruitment Status : Enrolling by invitation
First Posted : February 16, 2018
Last Update Posted : May 15, 2019
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment
Genetic Disorder Noonan Syndrome Drug: Somatropin

Layout table for study information
Study Type : Observational
Estimated Enrollment : 60 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
Actual Study Start Date : February 26, 2018
Estimated Primary Completion Date : November 30, 2021
Estimated Study Completion Date : November 30, 2021


Group/Cohort Intervention/treatment
Norditropin® (naïve participants)
The treatment period of Norditropin® for naïve participants will be up to 208 weeks.
Drug: Somatropin
Participants will be treated with commercially available Norditropin® (somatropin) according to routine clinical practice at the discretion of the treating physician.

Norditropin® (non-naïve participants)
The treatment period of Norditropin® for non-naïve participants will be up to 442 weeks.
Drug: Somatropin
Participants will be treated with commercially available Norditropin® (somatropin) according to routine clinical practice at the discretion of the treating physician.




Primary Outcome Measures :
  1. Number of adverse drug reactions (ADR) [ Time Frame: Weeks 0-208 ]
    Count of events


Secondary Outcome Measures :
  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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   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.
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03435627


Locations
Layout table for location information
Japan
Novo Nordisk Investigational Site
Kanagawa, Japan, 232-8555
Novo Nordisk Investigational Site
Kyoto, Japan, 602-8566
Novo Nordisk Investigational Site
Kyoto, Japan, 629-2261
Novo Nordisk Investigational Site
Maebashi-shi, Gunma, Japan, 371-8511
Novo Nordisk Investigational Site
Osaka, Japan, 594-1101
Novo Nordisk Investigational Site
Tochigi, Japan, 329-0498
Novo Nordisk Investigational Site
Tokyo, Japan, 157 8535
Novo Nordisk Investigational Site
Tokyo, Japan, 162-8666
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Layout table for investigator information
Study Director: Clinical Reporting Anchor and Disclosure (1452) Novo Nordisk A/S

Layout table for additonal information
Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT03435627     History of Changes
Other Study ID Numbers: GHLIQUID-4358
U1111-1191-3084 ( Other Identifier: World Health Organization (WHO) )
First Posted: February 16, 2018    Key Record Dates
Last Update Posted: May 15, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
URL: http://novonordisk-trials.com

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Genetic Diseases, Inborn
Disease
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Endocrine System Diseases
Cardiovascular Diseases
Heart Diseases
Connective Tissue Diseases
Dwarfism
Noonan Syndrome
Syndrome
Pathologic Processes
Craniofacial Abnormalities
Musculoskeletal Abnormalities
Heart Defects, Congenital
Cardiovascular Abnormalities
Congenital Abnormalities