Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome

NCT ID: NCT01927861

Last Updated: 2020-08-10

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

51 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-08-19

Study Completion Date

2018-07-12

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This trial is conducted in Asia. The aim of the trial is to investigate the long-term efficacy and safety of two doses of NN-220 (somatropin) in short stature due to Noonan syndrome.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Genetic Disorder Noonan Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

0.033 mg/kg/day

Group Type EXPERIMENTAL

somatropin

Intervention Type DRUG

Administered subcutaneously (s.c., under the skin) in a daily regimen for at least 104 weeks. Subject will be offered to continue treatment for another 104 weeks.

0.066 mg/kg/day

Group Type EXPERIMENTAL

somatropin

Intervention Type DRUG

Administered subcutaneously (s.c., under the skin) in a daily regimen for at least 104 weeks. Subject will be offered to continue treatment for another 104 weeks.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

somatropin

Administered subcutaneously (s.c., under the skin) in a daily regimen for at least 104 weeks. Subject will be offered to continue treatment for another 104 weeks.

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Japanese children with Noonan syndrome clinically diagnosed in one of the following ways: 1. Clinically diagnosed by at least two medical experts using van der Burgt score list, 2. Clinically diagnosed by one medical expert using van der Burgt score list and diagnosed by result of genetic testing for Noonan syndrome, 3. Clinically diagnosed by one medical expert using van der Burgt score list and diagnosed by the same medical expert based on the results of centralised evaluation of facial change using van der Burgt score list
* Height SDS (standard deviation score): -2 SDS or below (according to the Japanese reference data)
* Age: boys 3 to below 11 years, girls 3 to below 10 years
* Height records must be available within the period between 40 and 64 weeks prior to Visit 1 (screening)
* Prepubertal children (definition for girls breast and pubes of Tanner stage is I, and none of menses, and for boys testicular volume below 4 mL, and pubes and penis of Tanner stage is I)

Exclusion Criteria

* Children with known or suspected hypersensitivity against human growth hormone (hGH) or related products (including any components of the trial products)
* Children with diabetic type diagnosed with the Japanese Diabetes Society Classification
* Children with history or presence of active malignancy
* Children who have received GH (growth hormone) treatment
* Children who have received systemic administration of the following medications within two years prior to Visit 1 (screening): Thyroid hormone (except replacement therapy), antithyroid hormone, androgen, oestrogen, progesterone, anabolic steroid, adrenocortical steroid treatment period for at least 13 weeks), derivative of gonadotropin releasing hormone and somatomedin C (IGF-I)
Minimum Eligible Age

3 Years

Maximum Eligible Age

10 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Novo Nordisk A/S

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Global Clinical Registry (GCR, 1452)

Role: STUDY_DIRECTOR

Novo Nordisk A/S

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Novo Nordisk Investigational Site

Asahikawa, Hokkaido, , Japan

Site Status

Novo Nordisk Investigational Site

Fukuoka, , Japan

Site Status

Novo Nordisk Investigational Site

Fukuoka, , Japan

Site Status

Novo Nordisk Investigational Site

Iruma-gun, Saitama, , Japan

Site Status

Novo Nordisk Investigational Site

Kanagawa, , Japan

Site Status

Novo Nordisk Investigational Site

Kanagawa, , Japan

Site Status

Novo Nordisk Investigational Site

Kyoto, , Japan

Site Status

Novo Nordisk Investigational Site

Maebashi-shi, Gunma, , Japan

Site Status

Novo Nordisk Investigational Site

Miyazaki, , Japan

Site Status

Novo Nordisk Investigational Site

Nagoya, Aichi, , Japan

Site Status

Novo Nordisk Investigational Site

Niigata-shi, Niigata, , Japan

Site Status

Novo Nordisk Investigational Site

Osaka, , Japan

Site Status

Novo Nordisk Investigational Site

Osaka, , Japan

Site Status

Novo Nordisk Investigational Site

Ōita, , Japan

Site Status

Novo Nordisk Investigational Site

Saitama-city, Saitama, , Japan

Site Status

Novo Nordisk Investigational Site

Saitama-shi, Saitama, , Japan

Site Status

Novo Nordisk Investigational Site

Sapporo, Hokkaido, , Japan

Site Status

Novo Nordisk Investigational Site

Sendai-shi, Miyagi, , Japan

Site Status

Novo Nordisk Investigational Site

Shizuoka, , Japan

Site Status

Novo Nordisk Investigational Site

Tochigi, , Japan

Site Status

Novo Nordisk Investigational Site

Tokyo, , Japan

Site Status

Novo Nordisk Investigational Site

Tokyo, , Japan

Site Status

Novo Nordisk Investigational Site

Tokyo, , Japan

Site Status

Novo Nordisk Investigational Site

Tokyo, , Japan

Site Status

Novo Nordisk Investigational Site

Tokyo, , Japan

Site Status

Novo Nordisk Investigational Site

Tokyo, , Japan

Site Status

Novo Nordisk Investigational Site

Zentsuji, Kagawa, , Japan

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Japan

References

Explore related publications, articles, or registry entries linked to this study.

Horikawa R, Ogata T, Matsubara Y, Yokoya S, Ogawa Y, Nishijima K, Endo T, Ozono K. Long-term efficacy and safety of two doses of Norditropin(R) (somatropin) in Noonan syndrome: a 4-year randomized, double-blind, multicenter trial in Japanese patients. Endocr J. 2020 Aug 28;67(8):803-818. doi: 10.1507/endocrj.EJ19-0371. Epub 2020 May 9.

Reference Type RESULT
PMID: 32269181 (View on PubMed)

Ozono K, Ogata T, Horikawa R, Matsubara Y, Ogawa Y, Nishijima K, Yokoya S. Efficacy and safety of two doses of Norditropin(R) (somatropin) in short stature due to Noonan syndrome: a 2-year randomized, double-blind, multicenter trial in Japanese patients. Endocr J. 2018 Feb 26;65(2):159-174. doi: 10.1507/endocrj.EJ17-0313. Epub 2017 Nov 7.

Reference Type RESULT
PMID: 29109363 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

Access external resources that provide additional context or updates about the study.

http://novonordisk-trials.com

Clinical Trials at Novo Nordisk

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

U1111-1131-5892

Identifier Type: OTHER

Identifier Source: secondary_id

JapicCTI-132336

Identifier Type: REGISTRY

Identifier Source: secondary_id

GHLIQUID-4020

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.