Clinical trial NCT00184717: Growth Hormone Treatment in Children Born Small for Gestational Age (SGA)
Official title: GHLIQUID-1516: A 104-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 Subjects With Short Stature Born Small for Gestational Age / GHLIQUID-1517: A Long-term, Multi-centre, Randomised, Controlled, Double-blind, Parallel-group Trial, Investigating the Efficacy and Safety of Two Doses of NN-220 in Subjects With Short Stature Born Small for Gestational Age
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Foetal Growth Problem
Small for Gestational Age
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2005-09-16
Enrollment: 98
Sex: ALL
Minimum age (years): 3
Maximum age (years): 8
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study is conducted in Japan. The aim of this trial is to assess the efficacy and safety of somatropin in children born small for gestational age (SGA) in Japan.

In the main period, subjects will receive either active treatment for 104 weeks (two dosing regimens) or no treatment for 52 weeks followed by an extension period where subjects who received active treatment for 104 weeks (two years) will continue with the same treatment for further 156 weeks (three years) while those subjects who received no treatment for 52 weeks (one year) will be randomised to receive two dosing regimens for 208 weeks (four years). In total, subjects participate in trial for 260 weeks (five years).

Main period is registered internally at Novo Nordisk as GHLIQUID-1516 while the extension period is registered as GHLIQUID-1517.


Eligibility criteria: Inclusion Criteria:

* For MAIN period (GHLIQUID-1516):
* Born small for gestational age (SGA) with birth weight and birth length below the 10th percentile for gestational age, and additional either birth length below or equal to -2.0 standard deviation score (SDS) or birth weight below or equal to -2.0 SDS for gestational age
* Growth failure with height at -2.0 SDS or below for chronological age (CA)
* Normal growth hormone (GH) production, defined as peak GH level \> 10 ng/mL in one GH provocation test
* For EXTENSION period (GHLIQUID-1517):
* Subjects who completed the main period
* Chronological age (CA) for boys at least 4 years, but maximum 11 years
* Chronological age (CA) for girls at least 4 years, but maximum 10 years

Exclusion Criteria:

* Subjects with diabetes mellitus
* Subjects suffering from malignancy
* Several medical conditions

Primary outcomes:
measure: Change in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Week 260 - Subjects Received NN220 Treatment for 5 Years
timeFrame: Week 0, week 260
measure: Change in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Week 208 - Subjects Received NN220 Treatment for 4 Years
timeFrame: Week 0, week 208
Secondary outcomes:
measure: Yearly Height Velocity SDS for Chronological Age - Subjects Received NN220 Treatment for 5 Years
timeFrame: Weeks 0-260
measure: Yearly Height Velocity SDS for Chronological Age - Subjects Received NN220 Treatment for 4 Years
timeFrame: Weeks 0-208
measure: Change in Bone Age (Left Hand X-Ray) at Week 260 - Subjects Received NN220 Treatment for 5 Years
timeFrame: Week 0, week 260
measure: Change in Bone Age (Left Hand X-Ray) at Week 208 - Subjects Received NN220 Treatment for 4 Years
timeFrame: Week 0, week 208
measure: Adverse Events - Subjects Received NN220 Treatment for 5 Years
timeFrame: Weeks 0-260
measure: Adverse Events - Subjects Received NN220 Treatment for 4 Years
timeFrame: Weeks 0-208
Other outcomes:
Locations:
city: Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: Tanaka T, Yokoya S, Seino Y, Togari H, Mishina J, Kappelgaard AM, Fujieda K. Long-term efficacy and safety of two doses of growth hormone in short Japanese children born small for gestational age. Horm Res Paediatr. 2011;76(6):411-8. doi: 10.1159/000334152. Epub 2011 Nov 29.
citation: Kappelgaard AM, Kiyomi F, Horikawa R, Yokoya S, Tanaka T. The impact of long-term growth hormone treatment on metabolic parameters in Japanese patients with short stature born small for gestational age. Horm Res Paediatr. 2014;81(4):272-9. doi: 10.1159/000358196. Epub 2014 Feb 11.
citation: Horikawa R, Tanaka T, Nishinaga H, Ogawa Y, Yokoya S. The influence of a long-term growth hormone treatment on lipid and glucose metabolism: a randomized trial in short Japanese children born small for gestational age. Int J Pediatr Endocrinol. 2016;2016:19. doi: 10.1186/s13633-016-0036-4. Epub 2016 Oct 26.