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
Information source: Merck KGaA
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Hypochondroplasia
Intervention: Recombinant human growth hormone (Somatropin) (Drug)
Phase: Phase 2
Status: Active, not recruiting
Sponsored by: Merck KGaA Official(s) and/or principal investigator(s): Michel Polak, MD, PhD, Principal Investigator, Affiliation: Endocrinologie Pédiatrique & INSERM U845, centre des maladies rares de la croissance, Hôpital Necker Enfants Malades
Summary
This study is conducted to describe the efficacy and safety of recombinant human growth
hormone (r-hGH) treatment Saizen® on children with hypochondroplasia.
Clinical Details
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
Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Height-Standard deviation score (SDS) of treated children with hypochondroplasia over recombinant human growth hormone (r-hGH) treatment duration
Secondary outcome: Growth velocity (SDS/year) of treated children with hypochondroplasia over r-hGH treatment durationBody proportions of treated children with hypochondroplasia over r-hGH treatment duration Genotype fibroblast growth factor receptor (FGFR3) of subjects Body composition of treated children with hypochondroplasia over r-hGH treatment duration Adverse event (AE) and serious adverse event (SAE) during the treatment and follow-up period
Eligibility
Minimum age: 3 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
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
Locations and Contacts
Endocrinologie Pédiatrique - centre des maladies rares de la croissance -Hôpital Necker Enfants Malades, Paris 75015, France
Additional Information
Starting date: June 2009
Last updated: July 20, 2015
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