A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)
Condition: Growth Hormone Deficiency, Pediatric; hGH (Human Growth Hormone); Endocrine System Diseases; Hormones; Pituitary Diseases
MeSH terms: Dwarfism, Pituitary; Pituitary Diseases; Endocrine System Diseases
Intervention: Once weekly subcutaneous injection of TransCon hGH; Once daily subcutaneous injection of Genotropin
Intervention description: Once weekly subcutaneous injection; Once daily subcutaneous injection
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 162.0
Sex: All
Minimum age: 3 Years
Maximum age: 12 Years
Healthy volunteers: No
Keywords: Human Growth Hormone; hGH; GHD; rHGH; Pediatric Growth Hormone Deficiency; Long Acting Growth Hormone; Somatropin; Prodrug; Growth Failure; Growth Hormone Replacement Therapy; Sustained Release; Sustained Release Growth Hormone; Growth Hormone Deficiency

Summary:

      A 52 week trial of TransCon hGH, a long-acting growth hormone product, versus human growth
      hormone therapy. TransCon hGH will be given once-a-week, human growth hormone (hGH) will be
      given daily. Approximately 150 prepubertal, hGH-treatment naïve children (males and females)
      with GHD will be included. Randomization will occur in a 2:1 ratio (TransCon hGH :
      Genotropin). This is a global trial that will be conducted in Armenia, Australia, Belarus,
      Bulgaria, Georgia, Greece, Italy, New Zealand, Poland, Romania, Russia, Turkey, Ukraine, and
      the United States.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Prepubertal children with GHD (either isolated or as part of a multiple pituitary
             hormone deficiency) in Tanner stage 1 (Tanner 1982) aged:

               -  Boys: 3-12 years, inclusive

               -  Girls: 3-11 years, inclusive

          -  Impaired height (HT) defined as at least 2.0 standard deviations (SD) below the mean
             height for chronological age and sex (HT SDS ≤ -2.0) according to the 2000 CDC Growth
             Charts for the United States Methods and Development, available at
             http://www.cdc.gov/growthcharts/

          -  Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH
             level of ≤10 ng/mL, determined with a validated assay

          -  Bone age (BA) at least 6 months less than chronological age

          -  Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age
             and sex (IGF-1 SDS ≤-1)

          -  Written, signed informed consent of the parent(s) or legal guardian(s) of the subject
             and written assent of the subject (if the subject is able to read, understand, and
             sign)

        Exclusion Criteria:

          -  Children with a body weight below 12 kg

          -  Prior exposure to recombinant hGH or IGF-1 therapy

          -  Children with past or present intracranial tumor growth as confirmed by a sellar MRI
             scan (with contrast) at Screening (MRI results from up to 6 months prior to Screening
             may be accepted)

          -  Children with psychosocial dwarfism

          -  Children with idiopathic short stature

          -  History or presence of malignant disease; any evidence of present tumor growth

          -  Closed epiphyses

          -  Major medical conditions and/or presence of contraindication to hGH treatment

          -  Participation in any other trial of an investigational agent within 3 months prior to
             Screening