Clinical Trial: Cool.Click™ Adolescent Transition Study: Study of Saizen® in Subjects With Childhood-onset Growth Hormone Deficiency

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase IIIb, Prospective, Multicenter, Randomized, Open-label Study to Determine the Safety and Efficacy of Two Different Dosing Regimens of Saizen® (Recombinant Human Growth Hormone (r-hGH), Us

Brief Summary: The primary objective is to evaluate the efficacy and safety of two different dose regimens of r-hGH (Saizen®) in subjects with childhood-onset growth hormone deficiency (COGHD) during the transition phase from childhood to adulthood.

Detailed Summary:

This is a phase IIIb, prospective, multicenter, randomised, open label study to determine the safety and efficacy of two different dose regimens of r-hGH with a dose escalation scheme. Screening assessments must be completed 30 days prior to SD1 (Study Day 1). Eligible subjects ages 13 to 25 years will be randomised in equal allocation in a 1:1 ratio to one of two treatment groups (30 subjects/group). Daily subcutaneous injections will be self-administered or received from a designated individual using cool.click™, the needle-free growth hormone (GH) delivery device. The study consists of three periods: screening (up to 30 days prior to Study Day 1), active treatment (up to 24 weeks), and follow-up (4 week safety evaluation after the last dose of study medication).

Each subject will be required to complete a daily treatment diary to assess dosing compliance, adverse events, and concomitant medications. Each subject will receive one treatment diary at SD1, weeks 8, 12, and 24. Subjects will be required to record daily diary entries that will capture dosing compliance, adverse events, and concomitant medications. Depending upon treatment allocation and subject tolerability, dose titration will be increased as follows:

  • Group A: 0.005 mg/kg/day for 30 days then increasing, with the Investigator's approval, to 0.010 mg/kg/day from day 31 to week 24.
  • Group B: 0.010 mg/kg/day for 14 days with the opportunity to dose escalate, with the Investigator's approval, on day 15 to 0.02 mg/kg/day and day 29 to 0.03 mg/kg/day.

Scheduled study visits include screening, baseline, and weeks 8, 12, and 24. Dosage adjustments will be based on subject tolerability and telephone assessments from study drug initiation th
Sponsor: EMD Serono

Current Primary Outcome: Percent Change From Baseline to Week 24 in Trunk Fat [ Time Frame: Baseline to Week 24 ]

Original Primary Outcome: Increase in percent of trunk fat in COGHD

Current Secondary Outcome:

  • Percent Change From Baseline to Week 24 in Lean Body Mass [ Time Frame: Baseline to Week 24 ]
  • Percent Change From Baseline to Week 24 in Total Body Fat [ Time Frame: Baseline to Week 24 ]
  • Percent Change From Baseline to Week 24 in Limb Fat [ Time Frame: Baseline to Week 24 ]
  • Percent Change From Baseline to Week 24 in Trunk to Limb Fat Ratio [ Time Frame: Baseline to Week 24 ]


Original Secondary Outcome: Changes in lean body mass and body composition parameters

Information By: EMD Serono

Dates:
Date Received: May 2, 2005
Date Started: January 2005
Date Completion:
Last Updated: August 4, 2013
Last Verified: August 2013