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
Intervention: recombinant human growth hormone; recombinant human growth hormone
Intervention description: 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.
Intervention MeSH terms: Hormones
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 31.0
Sex: All
Minimum age: 13 Years
Maximum age: 25 Years
Healthy volunteers: No
Keywords: Childhood-onset growth hormone deficiency (COGHD); Saizen®

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.
    

Eligibility criteria:

        Inclusion Criteria:

        The day of entry or Study Day 1 is defined as the first day of study treatment. To be
        eligible for inclusion into this study, the subjects must fulfill all of the following
        criteria within 30 days prior to Study Day 1.

          -  Male or female from 13 to 25 years of age, inclusive

          -  Diagnosis of childhood onset growth hormone deficiency (GHD) and prior completed
             growth hormone (GH) treatment as evidenced by bone age greater than 14 years for girls
             and 16 years for boys or no height increase > 0.5 cm in the 6 months prior to Screen.

          -  Have documented GH deficiency (acquired or idiopathic), established by a standard
             provocative test, such as insulin (<5 ng/mL) or growth hormone releasing hormone plus
             arginine (<9 ng/mL) at least 30 days after GH has been discontinued. If a subject is
             hypopituitary with two or more pituitary disorders and has a low IGF-1, the
             stimulation test does not need to be performed to confirm GHD.

          -  If hypopituitary, must have been on adequate replacement therapy (if required) of
             glucocorticosteroids, thyroid and sex hormones (hormone levels on replacement being in
             normal/mildly elevated range) for at least 6 months prior to Screen.

          -  Be willing and able to comply with the protocol for the duration of the study.

          -  Have given written informed consent before any study-related procedure not part of the
             subject's normal medical care, with the understanding that the subject may withdraw
             consent at any time without prejudice to future medical care.

          -  Female subjects of childbearing potential must use a hormonal contraceptive,
             intra-uterine device, diaphragm with spermicide or condom with spermicide for the
             duration of the study. Confirmation that a female patient is not pregnant must be
             established by a negative human chorionic gonadotrophin (hCG) pregnancy test (urine or
             serum) within 7 days of study enrolment (SD1).

        Exclusion Criteria:

        To be eligible for inclusion in this study the subjects must not meet any of the following
        criteria:

          -  Known allergy or hypersensitivity to growth hormone or diluent.

          -  Previous treatment with GH within six months prior to Screen.

          -  Severe illness during the previous six months.

          -  Active malignancy (except non-melanomatous skin malignancies).

          -  Diabetes mellitus (type I or II).

          -  Seropositivity for human immunodeficiency virus (HIV), Hepatitis B surface antigen
             (HbsAg) and/or Hepatitis C Virus (HCV) serology.

          -  Pregnancy or lactation.

          -  History of drug and/or alcohol abuse or use of drugs for non-therapeutic purposes.

          -  Any medical condition that, in the opinion of the Investigator, would jeopardize the
             patient's safety following exposure to study drug.

          -  Clinically significant abnormal hematology, chemistry or urinalysis results at
             screening in the judgment of the Investigator.

          -  Have taken another investigational drug or had any experimental procedure in the six
             months preceding study entry.