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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at P25-P50 for all trials

Timeline

Completed

Started Feb 2018

Longer than P75 for all trials

Geographic Reach

1 country

22 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

3.9 years study duration

First Submitted

Initial submission to the registry

January 31, 2018

Completed

19 days until next milestone

First Posted

Study publicly available on registry

February 19, 2018

Completed

7 days until next milestone

Study Start

First participant enrolled

February 26, 2018

Completed

3.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 18, 2022

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 18, 2022

Completed

Last Updated

November 14, 2022

Status Verified

November 1, 2022

Enrollment Period

3.9 years

First QC Date

January 31, 2018

Last Update Submit

November 10, 2022

Conditions

Genetic Disorder Noonan Syndrome

Outcome Measures

Primary Outcomes (1)

Number of adverse drug reactions (ADR)
Count of events
Weeks 0-208

Secondary Outcomes (43)

Number of serious adverse drug reaction (SADR)
Week 0-208
Number of serious adverse event (SAE)
Week 0-208
Number of cardiac adverse event (AE)
Week 0-208
Change in insulin like growth factor I (IGF-I) for non-naïve patients
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
Weeks 0, 52, 104, 156, and 208 weeks
+38 more secondary outcomes

Study Arms (2)

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

SomatropinDRUG

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

Norditropin® (naïve participants)Norditropin® (non-naïve participants)

Eligibility Criteria

Age3 Years+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Sampling MethodNon-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.

You may qualify if:

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.

You may not qualify if:

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.

Contact the study team to confirm eligibility.