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

November 10, 2022 updated by: Novo Nordisk A/S
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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
71

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Japan
      • Asahikawa, Hokkaido, Japan, 078-8510
        • Novo Nordisk Investigational Site
      • Fukuoka, Japan, 830-0011
        • Novo Nordisk Investigational Site
      • Kanagawa, Japan, 216-8511
        • 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
      • Miyazaki, Japan, 889-1692
        • Novo Nordisk Investigational Site
      • Nagoya, Aichi, Japan, 467-8602
        • Novo Nordisk Investigational Site
      • Niigata-shi, Niigata, Japan, 951 8520
        • Novo Nordisk Investigational Site
      • Oita, Japan, 879-5593
        • Novo Nordisk Investigational Site
      • Osaka, Japan, 534-0021
        • Novo Nordisk Investigational Site
      • Osaka, Japan, 594-1101
        • Novo Nordisk Investigational Site
      • Saitama-shi, Saitama, Japan, 330-8777
        • Novo Nordisk Investigational Site
      • Sapporo, Hokkaido, Japan, 065-8611
        • Novo Nordisk Investigational Site
      • Sendai-shi, Miyagi, Japan, 980 8574
        • Novo Nordisk Investigational Site
      • Shizuoka, Japan, 431-3192
        • Novo Nordisk Investigational Site
      • Tochigi, Japan, 329-0498
        • Novo Nordisk Investigational Site
      • Tokyo, Japan, 157 8535
        • Novo Nordisk Investigational Site
      • Tokyo, Japan, 160-8582
        • Novo Nordisk Investigational Site
      • Tokyo, Japan, 162-8666
        • Novo Nordisk Investigational Site
      • Tokyo, Japan, 183-8561
        • Novo Nordisk Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

3 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of adverse drug reactions (ADR)
Time Frame: Weeks 0-208
Count of events
Weeks 0-208

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Number of serious adverse drug reaction (SADR)
Time Frame: Week 0-208
Count of events
Week 0-208
Number of serious adverse event (SAE)
Time Frame: Week 0-208
Count of events
Week 0-208
Number of cardiac adverse event (AE)
Time Frame: Week 0-208
Count of events
Week 0-208
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
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
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
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
Time Frame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
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
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Presented as SD score
Weeks 0, 52, 104, 156, and 208 weeks
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 %
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in haemoglobin A1c (HbA1c) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Measured in %
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in aspartate aminotransferase (AST) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Measured in IU/L
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in alanine aminotransferase (ALT) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Measured in IU/L
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in total cholesterol (T-CHO) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Measured in mg/dL
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
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
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
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
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in triglyceride (TG) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Measured in mg/dL
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in thyroid stimulation hormone(TSH) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Measured in μU/mL
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in free triiodothyronine (FT-3) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Measured in pg/mL
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in free thyroxine (FT-4) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Measured in ng/mL
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in white blood cell (WBC) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Measured in μ/L
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in platelet (PLT) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Measured in 10^4/μL
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in bone age (BA) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Measured in year
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in bone age/chronological age (BA/CA) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Presented as ratio
Weeks 0, 52, 104, 156, and 208 weeks
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.
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
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.
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Change in height standard deviation score (HSDS) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Presented as SD score
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Height velocity standard deviation score (HVSDS) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Presented as SD score
Weeks 0, 52, 104, 156, and 208 weeks
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
Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
Height velocity (HV) for naïve patients
Time Frame: Weeks 0, 52, 104, 156, and 208 weeks
Measured in cm/year
Weeks 0, 52, 104, 156, and 208 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novo Nordisk A/S

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 26, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 18, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 18, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 31, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 9, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 19, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 14, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 10, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GHLIQUID-4358
  • U1111-1191-3084 (Other Identifier: World Health Organization (WHO))

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Yes

IPD Plan Description

According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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