Clinical trial NCT00249821: Optimization of the Dosage Regimen of Growth Hormone Therapy in Children Born Small for Gestational Age
Official title: Optimization of the Dosage Regimen With Growth Hormone Therapy in Children Born Small for Gestational Age. An Open Label, Randomized, Pilot Study, Comparing in Children Treated for 3 Years, the Efficacy of a Saizen® Treatment at the Same Dose Versus a Lower Maintenance Dose Prolonged During 1 Additional Year.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Small Gestational Age (SGA)
Lead sponsor: Merck KGaA, Darmstadt, Germany
Sponsor class: INDUSTRY
Collaborators: name: Merck Serono S.A.S., an affiliate of Merck KGaA, Darmstadt, Germany
First posted: 2005-11-07
Enrollment: 22
Sex: ALL
Minimum age (years): 6
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Multicentric, open-label, randomized, pilot comparative study in parallel groups comparing 1 group of subjects receiving 0.057 milligram/kilogram/day (mg/kg/day) or 0.40 mg/kg/week of Saizen® during 1 year to 1 group receiving 0.035 mg/kg/day (0.24 mg/kg/week) of Saizen® during 1 year after an initial 3-year treatment of recombinant human growth hormone (r-hGH) therapy with 0.057 mg/kg/day in both groups.


Eligibility criteria: Inclusion Criteria:

To be eligible for inclusion / randomization into this study, the subjects must fulfill all of the following criteria (if there is no inclusion phase, the inclusion criteria will be considered as inclusion criteria for randomization):

* Written consent form signed by the parents / legal guardian, and child if possible
* Subject born SGA and receiving a r-hGH therapy for this pathology
* Recombinant human growth hormone (r-hGH) started at the maximal chronological age of 7 years for girls and 8 years for boys
* Treatment with r-hGH started for at least 30 months and less than 36 months at 0.057 mg/kg/day
* Height gain during the first 2 years of GH treatment \> 1 SD compared with the initial value

Exclusion Criteria:

To be eligible for inclusion in this study the subjects must not meet any of the following criteria:

* Known hypersensitivity to Somatropin or any of the excipients
* Active neoplasia (either newly diagnosed or recurrent)
* Intracranial hypertension
* Known diabetes mellitus
* Proliferative or preproliferative diabetic retinopathy
* Evidence of any progression or recurrence of an underlying intra-cranial space occupying lesion
* Obesity defined as degree 1 on the corpulence curves
* Precocious puberty
* Pubertal status: Tanner breast development stage \> 2 for girls, and testicular volume \> 4 milliliter (mL) or testicular length \> 3 centimeter (cm) and/or testosterone value \>1 nanomole/liter \[nmol/L\] (0.29 gram/mL \[g/mL\]) for boys For girls \> 9 years and Tanner breast development stage 1: uterine size \> 35 millimeter (mm)
* Severe chronic concomitant illness such as chronic renal failure, cystic fibrosis
* Concomitant corticoid treatment or levothyroxine treatment other than substitutive treatment, topical or inhaled treatment
* Participation to any clinical study within the 30 days preceding study entry

Primary outcomes:
measure: Height Velocity
timeFrame: Month 12
Secondary outcomes:
measure: Change From Baseline in Height-Standard Deviation Score (H-SDS) at Month 6 and Month 12
timeFrame: Baseline (randomization), Month 6 and Month 12
measure: Height Velocity-Standard Deviation Score (HV-SDS)
timeFrame: Month 6 and Month 12
measure: Change From Baseline in Height at Month 6
timeFrame: Baseline (randomization) and Month 6
measure: Change From Baseline in Bone Age at Month 12
timeFrame: Baseline (randomization) and Month 12
measure: Insulin Like Growth Factor-1 (IGF-1) Levels
timeFrame: Baseline (randomization), Month 6 and Month 12
measure: Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels
timeFrame: Baseline (randomization), Month 6 and Month 12
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
timeFrame: Baseline (randomization) until Month 12
Other outcomes:
Locations:
Study officials:
affiliation: Merck Serono S.A.S, France
name: Medical Responsible
role: STUDY_DIRECTOR
References:
citation: Chatelain P, Colle M, Nako JP, Le Luyer B, Wagner K, Berlier P, Tauber M. Optimization of growth hormone dosing in children born small for gestational age: an open-label, randomized study of children during the fourth year of therapy. Horm Res Paediatr. 2012;77(3):156-63. doi: 10.1159/000337216. Epub 2012 Apr 12.