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

Merck KGaA (EMD Serono)

Status and phase

Completed

Phase 2

Conditions

Hypochondroplasia

Treatments

Drug: Recombinant human growth hormone (r-hGH)

Study type

Interventional

Funder types

Industry

Identifiers

NCT01111019

IMP 26545 (EMR701048-506)

Details and patient eligibility

About

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

Enrollment

19 patients

Sex

All

Ages

3+ years old

Volunteers

No Healthy Volunteers

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