Optimization of the Dosage Regimen of Growth Hormone Therapy in Children Born Small for Gestational Age (SGA OPTIMIS)

Merck KGaA (EMD Serono)

Status and phase: Completed; Phase 3

Conditions: Small Gestational Age (SGA)

Treatments: Drug: Saizen®

Study type: Interventional

Funder types: Industry; Other

Identifiers

NCT00249821

IMP 25735

Details and patient eligibility

About

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.

Enrollment: 22 patients

Sex: All

Ages: 6 to 11 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

22 participants in 2 patient groups

Saizen® 0.057 mg/kg/day

Experimental group

Description:

Subjects who met all inclusion/exclusion criteria were randomly assigned to 0.057 mg/kg/day in this multi-center study. Subjects were stratified at randomization according to the Height-Standard Deviation Score (H-SDS) at this time (less than \[\<\] -2 SDS or greater than \[\>\] -2 SDS)

Treatment:

Drug: Saizen®

Drug: Saizen®

Saizen® 0.035 mg/kg/day

Experimental group

Description:

Subjects who met all inclusion/exclusion criteria were randomly assigned to 0.035 mg/kg/day in this multi-center study. Subjects were stratified at randomization according to the H-SDS at this time (\< -2 SDS or \> -2 SDS)

Treatment:

Drug: Saizen®

Drug: Saizen®