Overview

This trial is active, not recruiting.

Conditions genetic disorder, noonan syndrome
Treatment somatropin
Phase phase 3
Sponsor Novo Nordisk A/S
Start date August 2013
End date January 2017
Trial size 51 participants
Trial identifier NCT01927861, GHLIQUID-4020, JapicCTI-132336, U1111-1131-5892

Summary

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator)
Primary purpose treatment
Arm
(Experimental)
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.
(Experimental)
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.

Primary Outcomes

Measure
Change in height SDS (Standard Deviation Score)
time frame: Week 0, week 104
Change in height SDS (Standard Deviation Score)
time frame: Week 0, week 208

Secondary Outcomes

Measure
Incidence of treatment emergent adverse events (AEs)
time frame: Weeks 0, Week 104
Incidence of treatment emergent adverse events (AEs)
time frame: Weeks 0, Week 208
Change in IGF-I (insulin like growth factor I)
time frame: Week 0, week 104
Change in IGF-I (insulin like growth factor I)
time frame: Week 0, week 208
Change in HbA1c (Glycosylated haemoglobin)
time frame: Week 0, week 104
Change in HbA1c (Glycosylated haemoglobin)
time frame: Week 0, week 208

Eligibility Criteria

Male or female participants from 3 years up to 10 years old.

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)

Additional Information

Official title A 52-week, Multi-centre, Randomised, Double-blind, Parallel-group, no Treatment Controlled (Open-label) Trial Investigating the Efficacy and Safety of Two Doses of NN-220 in Short Stature With Noonan Syndrome
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Novo Nordisk A/S.