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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 duration

Body 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

Page last updated: August 23, 2015

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