Overview

This trial is active, not recruiting.

Condition hypochondroplasia
Treatment recombinant human growth hormone (somatropin)
Phase phase 2
Sponsor Merck KGaA
Collaborator Merck Serono S.A.S, France
Start date June 2009
End date April 2018
Trial size 9 participants
Trial identifier NCT01111019, IMP 26545 (EMR701048-506)

Summary

This study is conducted to describe the efficacy and safety of recombinant human growth hormone (r-hGH) treatment Saizen® on children with hypochondroplasia.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Subjects will be treated with 0.057 milligram per kilogram per day (mg/kg/day) r-hGH. Subjects still under treatment after 31 January 2011 will be treated with a dose reduced to 0.035 mg/kg/day until the end of the study.
recombinant human growth hormone (somatropin) Saizen®
Recombinant human growth hormone (Somatropin) will be administered at 0.057 mg/kg/day subcutaneous injection for 3 years, 5 years or until near final height is reached, if applicable. Subjects still under treatment after 31 January 2011 will be treated with a dose reduced to 0.035 mg/kg/day until the end of the study.
(No Intervention)

Primary Outcomes

Measure
Height-Standard deviation score (SDS) of treated children with hypochondroplasia over recombinant human growth hormone (r-hGH) treatment duration
time frame: 3 years to 5 years or until near final height is reached, (if applicable)

Secondary Outcomes

Measure
Growth velocity (SDS/year) of treated children with hypochondroplasia over r-hGH treatment duration
time frame: 3 years to 5 years or until near final height is reached, (if applicable)
Body proportions of treated children with hypochondroplasia over r-hGH treatment duration
time frame: 3 years to 5 years or until near final height is reached, (if applicable)
Genotype fibroblast growth factor receptor (FGFR3) of subjects
time frame: 3 to 5 years (if applicable)
Body composition of treated children with hypochondroplasia over r-hGH treatment duration
time frame: 3 years to 5 years or until near final height is reached, (if applicable)
Adverse event (AE) and serious adverse event (SAE) during the treatment and follow-up period
time frame: years to 5 years or until near final height is reached, (if applicable)

Eligibility Criteria

Male or female participants at least 3 years old.

Inclusion Criteria: - Male or female children with hypochondroplasia defined by a disproportional short limb height and a X-ray evidence of shortening of the long bones and failure of increase in the interpedicular distance between lumbar vertebrae L1 and L5 - Result of genetic analysis for mutation of gene FGFR3 already known or ongoing analysis at the beginning of the study - Chronological age greater than or equal to 3 years - Height for chronological age less than or equal to - 2 SDS - Bone age less than or equal to 11 years for girls and 13 years for boys - A written informed consent at the beginning of the pre-treatment period must be obtained from the parent(s)/legal guardian(s). Children able to understand the trial should personally sign and date the written informed consent Additional inclusion criteria for each study prolongation: - Bone age at Month 36 or Month 60 is compatible with treatment prolongation according to investigator opinion - Subject is still under r-hGH treatment with Saizen® at Month 36 or Month 60 - Height gain greater than or equal to + 1 SDS after the 2 first years of treatment for treatment prolongation at Month 36 and growth velocity greater than or equal to 5 centimeter (cm) per year, with bone age less than 14 years for females or less than 16 years for males for treatment prolongation at Month 60 - According to investigator opinion, gene mutations of the subjects are not in connection with observed side effects during the 3 or 5 first years of treatment - An updated written informed consent must be obtained from the parent(s)/legal guardian(s) before the start of each study prolongation. Children able to understand the trial should personally sign and date the written informed consent Exclusion Criteria: - Turner's Syndrome in girls - Active malignant neoplastic disease - Severe congenital malformations - Proliferative or preproliferative diabetic retinopathy - Evidence of any progression or recurrence of an underlying intra-cranial space occupying lesion - Severe psychomotor retardation - Diabetes mellitus or history of significant glucose intolerance as defined by a fasting blood glucose greater than 6.4 millimole per liter (mmol/L) - Known renal insufficiency as defined by serum creatinine level 1.0 milligram per deciliter (mg/dL) (88 micromole per liter [mcmol/L]) - Known hepatic disease as defined by elevated liver enzymes or total bilirubin (* 2 Normal) - Current congestive heart failure, untreated hypertension, serious chronic edema of any cause - Chronic infectious disease - History of intracranial hypertension with papilledema - Previous or ongoing treatment with sex steroid therapy such as estrogens or testosterone - Previous or ongoing treatment with any therapy that may directly influence growth, including Growth Hormone (GH), Growth Hormone Releasing Hormone (GHRH) and long duration corticosteroids therapy - Known hypersensitivity to somatropin or any of the excipients - Epiphyseal fusion - Participation to any clinical study within the 30 days preceding study entry - Pregnant females

Additional Information

Official title Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated Children With Hypochondroplasia
Principal investigator Michel Polak, MD, PhD
Trial information was received from ClinicalTrials.gov and was last updated in July 2015.
Information provided to ClinicalTrials.gov by Merck KGaA.