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

NCT00249821 | Completed Phase 3 Results

• 1 other identifier: IMP 25735
• Study Type: interventional
• Target: 22
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Small Gestational Age (SGA)

Outcome Measures

Primary Outcomes (1)

• Height Velocity

  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.

  Month 12

Secondary Outcomes (7)

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

  Baseline (randomization), Month 6 and Month 12

• Height Velocity-Standard Deviation Score (HV-SDS)

  Month 6 and Month 12

• Change From Baseline in Height at Month 6

  Baseline (randomization) and Month 6

• Change From Baseline in Bone Age at Month 12

  Baseline (randomization) and Month 12

• Insulin Like Growth Factor-1 (IGF-1) Levels

  Baseline (randomization), Month 6 and Month 12

Study Arms (2)

Saizen® 0.057 mg/kg/day

EXPERIMENTAL

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)

Drug: Saizen®

Saizen® 0.035 mg/kg/day

EXPERIMENTAL

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)

Drug: Saizen®

Interventions

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.

Also known as: somatropin

Saizen® 0.057 mg/kg/day

Eligibility Criteria

• Age: 6 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

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

Related Publications (1)

• 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.

  PMID: 22508151 RESULT

Related Links

• Related Info

MeSH Terms

Interventions

Human Growth Hormone

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Limitations and Caveats

Participants' inclusion period was extended but due to remaining low recruitment rate, it was finally decided to stop enrollment even if only 22 participants were enrolled.

Results Point of Contact

• Title: Merck KGaA Communication Center
• Organization: Merck Serono, a division of Merck KGaA

service@merckgroup.com

+49-6151-72-5200

Study Officials

• Medical Responsible

  Merck Serono S.A.S, France

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 4, 2005
• First Posted: November 7, 2005
• Study Start: February 28, 2005
• Primary Completion: September 30, 2007
• Study Completion: September 30, 2007
• Last Updated: September 26, 2017
• Results First Posted: October 10, 2012

Record last verified: 2017-08