Clinical Trial: 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
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
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
Brief Summary: This study is conducted to describe the efficacy and safety of recombinant human growth hormone (r-hGH) treatment Saizen® on children with hypochondroplasia.
Detailed Summary:
Sponsor: Merck KGaA
Current Primary Outcome: 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) ]
Original Primary Outcome: Height-Standard deviation score (SDS) of treated children with hypochondroplasia over recombinant human growth hormone (r-hGH) treatment duration [ Time Frame: 3 to 5 years (if applicable) ]
Current Secondary Outcome:
- 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) ]Body proportions include head circumference, superior segment, height, weight, and body mass index (BMI)
- 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) ]Body composition includes percent body fat, lean muscle mass, bone mineral density (BMD), osteocalcin, and carboxyterminal cross-linking (CTX)
- 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) ]This includes local tolerance at the injection site, clinical tolerance at each visit and assessment of biological tolerance every 6 months.
Original Secondary Outcome:
- The growth velocity (SDS/year) of treated children with hypochondroplasia over r-hGH treatment duration. [ Time Frame: 3 to 5 years (if applicable) ]
- The body proportions of treated children with hypochondroplasia over r-hGH treatment duration. [ Time Frame: 3 to 5 years (if applicable) ]Body proportions include head circumference, superior segment, height, weight, body mass index (BMI)
- The genotype fibroblast growth factor receptor (FGFR3) of subjects [ Time Frame: 3 to 5 years (if applicable) ]
- The body composition of treated children with hypochondroplasia over r-hGH treatment duration. [ Time Frame: 3 to 5 years (if applicable) ]Body composition includes percent body fat, lean muscle mass, bone mineral density (BMD), osteocalcin, CTX
- Adverse event (AE) and serious adverse event (SAE) during the treatment and follow-up period. [ Time Frame: 3 to 5 years (if applicable) ]This includes local tolerance at the injection site, clinical tolerance at each visit and assessment of biological tolerance every 6 months.
Information By: Merck KGaA
Dates:
Date Received: April 23, 2010
Date Started: June 2009
Date Completion: April 2018
Last Updated: January 3, 2017
Last Verified: January 2017