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Phase 3 N=22 Randomized Treatment

Optimization of the Dosage Regimen of Growth Hormone Therapy in Children Born Small for Gestational Age

Small Gestational Age (SGA)

Enrolled (actual)
22
Serious AEs
0.0%
Results posted
Oct 2012
Primary outcome: Primary: Height Velocity — 6.40; 4.40 centimeter (cm)/year — p=0.001

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Saizen® (Drug)
Age
Pediatric · 6+ yrs
Sex
All
Sponsor
Merck KGaA, Darmstadt, Germany
Primary completion
Sep 2007

Outcome Measures

OutcomeResultp-value
PRIMARY
Height Velocity
6.40; 4.40 0.001 sig
SECONDARY
Change From Baseline in Height-Standard Deviation Score (H-SDS) at Month 6 and Month 12
-1.30; -1.50; 0.30; 0.10; 0.30; -0.10 0.012 sig
SECONDARY
Height Velocity-Standard Deviation Score (HV-SDS)
0.60; 0.10; 0.30; -0.10 0.022 sig
SECONDARY
Change From Baseline in Height at Month 6
121.10; 119.20; 3.60; 2.50 0.029 sig
SECONDARY
Change From Baseline in Bone Age at Month 12
7.50; 7.00; 1.40; 1.40 0.972
SECONDARY
Insulin Like Growth Factor-1 (IGF-1) Levels
319.60; 275.40; 369.30; 262.40; 414.30; 263.00 0.058
SECONDARY
Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels
3.40; 3.40; 3.00; 3.30; 3.60; 3.50 0.793
SECONDARY
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
2; 1

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
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00249821). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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