Cool.Click™ Adolescent Transition Study: Study of Saizen® in Subjects With Childhood-onset Growth Hormone Deficiency
Condition: Childhood-onset Growth Hormone Deficiency; Pituitary Dwarfism
MeSH terms: Dwarfism, Pituitary; Dwarfism; Endocrine System Diseases
Study type: Interventional
Phase: Phase 3

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 description: 
      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 through week 6. Trunk fat will be measured at SD1, weeks 12 and 24 (or early
      termination visit). Routine clinical laboratory assessments (hematology, blood chemistries,
      and urinalysis) will be performed pre-treatment (-30 to -1 SD1) and post-treatment on week 24
      (or early termination visit). Special laboratory assessments include the central analysis of
      lipid panel, fasting insulin, fasting glucose, insulin-like growth factor I (IGF-I),
      insulin-like growth factor binding protein 3 (IGFBP-3), free thyroxine (T4) , total T4,
      C-reactive protein (CRP). Physical exams will be performed at screening, weeks 12 and 24.
      Safety evaluations will occur during scheduled study visits, through telephone assessments,
      and by the review of adverse events and concomitant events on the subject treatment diary.
    

Intervention name(s): recombinant human growth hormone; recombinant human growth hormone
Intervention type(s): Biological; Biological
Intervention description(s): 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.; 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.
Other intervention name(s): Saizen; r-hGH; cool click; Saizen; r-hGH; cool click
Intervention MeSH terms: Hormones
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Childhood-onset growth hormone deficiency (COGHD); Saizen®