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Completed

NCT Number: NCT00249821

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

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.

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Key information

Conditions

Small Gestational Age (SGA)

Age range

6 year–11 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Saizen®

Drug

Saizen® [somatropin (recombinant deoxyribonucleic acid [rDNA] origin) for injection], a r-hGH, is indicated for the treatment of children with growth failure due to inadequate secretion of endogenous growth hormone.

Other names: somatropin

Primary outcomes

  1. Height Velocity

    Time frame: Month 12

    Height Velocity (HV) is the change in height since the previous year´s measurement and more precisely: HV = {(h-hp)/(d-dp)} * 365.25 [centimeter (cm)/year] where h is current height in cm, hp is previous height in cm, closest to 1 year previous, d is the current date and dp is the date of measurement of previous height, closest to 1 year previous. Additionally, d and dp have to be within 0.6 years and 1.5 years. HV is the mean height velocity over the interval between d and dp but is displayed as HV at d.

Secondary outcomes

  1. Change From Baseline in Height-Standard Deviation Score (H-SDS) at Month 6 and Month 12

    Time frame: Baseline (randomization), Month 6 and Month 12

    Height-Standard Deviation Score (H-SDS) was calculated as height minus mean (age-and sex-matched reference) divided by standard deviation (SD) [age and sex-matched reference]. Greater H-SDS indicates greater height.

  2. Height Velocity-Standard Deviation Score (HV-SDS)

    Time frame: Month 6 and Month 12

    Height Velocity-Standard Deviation Score (HV-SDS) was calculated as height velocity minus reference mean height velocity divided by SD of the reference mean height velocity. Greater HV-SDS indicates greater height velocity.

  3. Change From Baseline in Height at Month 6

    Time frame: Baseline (randomization) and Month 6

  4. Change From Baseline in Bone Age at Month 12

    Time frame: Baseline (randomization) and Month 12

    Bone age was assessed by a left wrist X-Ray and evaluated by the investigator according to the Greulich and Pyle method.

  5. Insulin Like Growth Factor-1 (IGF-1) Levels

    Time frame: Baseline (randomization), Month 6 and Month 12

  6. Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels

    Time frame: Baseline (randomization), Month 6 and Month 12

  7. Number of Participants With Treatment Emergent Adverse Events (TEAEs)

    Time frame: Baseline (randomization) until Month 12

    Adverse Events (AEs): Any untoward medical occurrence in the form of signs, clinically significant abnormalities in laboratory findings, diseases, symptoms, or worsening of complications. TEAEs: AEs that occur during treatment with the Investigational Medicinal Product (IMP).

Sponsors and collaborators

Lead sponsor

Merck KGaA, Darmstadt, Germany

Industry

Collaborators

  • Merck Serono S.A.S., an affiliate of Merck KGaA, Darmstadt, Germany

Registry information

Official study 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.

Acronym: SGA OPTIMIS

Important dates

Study start
2005
Primary completion
2007
Study completion
2007
First posted
Nov 7, 2005
Registry last updated
Sep 26, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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