NCT01927861|CompletedPhase 3Results

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

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

Novo Nordisk A/S ClinicalTrials.gov

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3 other identifiers

GHLIQUID-4020U1111-1131-5892JapicCTI-132336

Study Type

interventional

Target

Locations

1 country

Sites

Timeline

RegisteredAug 2013

StartedAug 2013

CompletionJul 2018

Brief 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.

Trial Health

On Track

Trial Health Score

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

Enrollment

participants targeted

Target at below P25 for phase_3

Timeline

Completed

Started Aug 2013

Longer than P75 for phase_3

Geographic Reach

1 country

27 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

4.9 years study duration

Study Start

First participant enrolled

August 19, 2013

Completed

1 day until next milestone

First Submitted

Initial submission to the registry

August 20, 2013

Completed

3 days until next milestone

First Posted

Study publicly available on registry

August 23, 2013

Completed

4.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 12, 2018

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 12, 2018

Completed

1.3 years until next milestone

Results Posted

Study results publicly available

October 14, 2019

Completed

Last Updated

August 10, 2020

Status Verified

July 1, 2020

Enrollment Period

4.9 years

First QC Date

August 20, 2013

Results QC Date

July 4, 2019

Last Update Submit

July 27, 2020

Conditions

Genetic Disorder Noonan Syndrome

Outcome Measures

Primary Outcomes (1)

Change in Height SDS (Japanese National Reference Data)
Height SDS was calculated using the formula: SDS = (height - mean)/SD, where height was the height variable measured, mean and SD of height by sex and age for the reference population. The scores were centered around zero. Positive SDS indicated greater height and negative SDS indicated lesser height than the mean of the reference population. The change from baseline (week 0) in the height SDS after 104 weeks of treatment was analysed using an analysis of covariance (ANCOVA) model with treatment as a fixed effect and baseline height SDS as a covariate. Positive value in change from baseline indicated that SDS was better than baseline SDS. Missing values were imputed using the last observation carried forward (LOCF) method.
Baseline, week 104

Secondary Outcomes (66)

Height Velocity SDS
Baseline to week 52
Height Velocity SDS
Week 52 to week 104
Height Velocity
Baseline to week 52
Height Velocity
Week 52 to week 104
Incidence of Treatment Emergent Adverse Events
During 104 weeks of treatment
+61 more secondary outcomes

Study Arms (2)

0.033 mg/kg/day

EXPERIMENTAL

Drug: somatropin

0.066 mg/kg/day

EXPERIMENTAL

Drug: somatropin

Interventions

somatropinDRUG

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.

0.033 mg/kg/day0.066 mg/kg/day

Eligibility Criteria

Age3 Years - 10 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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)

You may not qualify if:

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)

Contact the study team to confirm eligibility.