Clinical trial NCT03435627: Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
Official title: Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Genetic Disorder
Noonan Syndrome
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2018-02-19
Enrollment: 71
Sex: ALL
Minimum age (years): 3
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.


Eligibility 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.

Primary outcomes:
measure: Number of adverse drug reactions (ADR)
timeFrame: Weeks 0-208
Secondary outcomes:
measure: Number of serious adverse drug reaction (SADR)
timeFrame: Week 0-208
measure: Number of serious adverse event (SAE)
timeFrame: Week 0-208
measure: Number of cardiac adverse event (AE)
timeFrame: Week 0-208
measure: Change in insulin like growth factor I (IGF-I) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in insulin like growth factor I (IGF-I) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in insulin like growth factor I standard deviation score (IGF-I SDS) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in insulin like growth factor I standard deviation score (IGF-I SDS) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in haemoglobin A1c (HbA1c) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in haemoglobin A1c (HbA1c) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in aspartate aminotransferase (AST) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in aspartate aminotransferase (AST) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in alanine aminotransferase (ALT) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in alanine aminotransferase (ALT) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in total cholesterol (T-CHO) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in total cholesterol (T-CHO) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in high-density lipoprotein cholesterol (HDL-C) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in high-density lipoprotein cholesterol (HDL-C) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in Low-density lipoprotein cholesterol (LDL-C) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in Low-density lipoprotein cholesterol (LDL-C) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in triglyceride (TG) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in triglyceride (TG) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in thyroid stimulation hormone(TSH) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in thyroid stimulation hormone(TSH) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in free triiodothyronine (FT-3) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in free triiodothyronine (FT-3) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in free thyroxine (FT-4) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in free thyroxine (FT-4) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in white blood cell (WBC) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in white blood cell (WBC) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in platelet (PLT) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in platelet (PLT) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in bone age (BA) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in bone age (BA) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in bone age/chronological age (BA/CA) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in bone age/chronological age (BA/CA) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in electrocardiogram (ECG) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in electrocardiogram (ECG) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Change in height standard deviation score (HSDS) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Change in height standard deviation score (HSDS) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Height velocity standard deviation score (HVSDS) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Height velocity standard deviation score (HVSDS) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
measure: Height velocity (HV) for non-naïve patients
timeFrame: Weeks 0, 52, 104, 156, 208, 260, 312, 364, 416, and 442 weeks
measure: Height velocity (HV) for naïve patients
timeFrame: Weeks 0, 52, 104, 156, and 208 weeks
Other outcomes:
Locations:
city: Asahikawa, Hokkaido
country: Japan
facility: Novo Nordisk Investigational Site
city: Fukuoka
country: Japan
facility: Novo Nordisk Investigational Site
city: Kanagawa
country: Japan
facility: Novo Nordisk Investigational Site
city: Kanagawa
country: Japan
facility: Novo Nordisk Investigational Site
city: Kyoto
country: Japan
facility: Novo Nordisk Investigational Site
city: Kyoto
country: Japan
facility: Novo Nordisk Investigational Site
city: Maebashi-shi, Gunma
country: Japan
facility: Novo Nordisk Investigational Site
city: Miyazaki
country: Japan
facility: Novo Nordisk Investigational Site
city: Nagoya, Aichi
country: Japan
facility: Novo Nordisk Investigational Site
city: Niigata-shi, Niigata
country: Japan
facility: Novo Nordisk Investigational Site
city: Oita
country: Japan
facility: Novo Nordisk Investigational Site
city: Osaka
country: Japan
facility: Novo Nordisk Investigational Site
city: Osaka
country: Japan
facility: Novo Nordisk Investigational Site
city: Saitama-shi, Saitama
country: Japan
facility: Novo Nordisk Investigational Site
city: Sapporo, Hokkaido
country: Japan
facility: Novo Nordisk Investigational Site
city: Sendai-shi, Miyagi
country: Japan
facility: Novo Nordisk Investigational Site
city: Shizuoka
country: Japan
facility: Novo Nordisk Investigational Site
city: Tochigi
country: Japan
facility: Novo Nordisk Investigational Site
city: Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
city: Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
city: Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
city: Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Reporting Anchor and Disclosure (1452)
role: STUDY_DIRECTOR
References: