rhGH and rhIGF-1 Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency
Condition: Insulin-like Growth Factor-1 Deficiency
MeSH terms: Dwarfism
Intervention: NutropinAq® (Somatropin [rDNA origin]); Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin]); Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin]); Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])
Intervention description: rhGH (Somatropin) 45µg/kg once daily injection; rhGH 45µg/kg and rhIGF-1 150µg/kg once daily injection; rhGH (Somatropin) 45µg/kg and rhIGF-1 (Mecasermin) 50µg/kg once daily injections; rhGH 45µg/kg and rhIGF-1 100µg/kg once daily injections
Intervention MeSH terms: Mecasermin
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 106.0
Sex: All
Minimum age: 5 Years
Healthy volunteers: No
Keywords: IGF-1 Deficiency; growth; ISS; Constitutional growth delay; Primary IGFD; Primary IGF Deficiency

Summary:

      IGF-1 (insulin-like growth factor-1) is a hormone that is normally produced in the body in
      response to another hormone called growth hormone. Growth Hormone is produced by a small
      gland at the base of the brain (the pituitary). Together IGF-1 and GH are large contributors
      to growth during infancy, childhood, and adolescence.

      Children with IGF Deficiency are short and have an imbalance in the levels of growth hormone
      and IGF-1 that their body produces. Their growth hormone levels are normal or even high, but
      IGF-1 levels do not increase normally in response to growth hormone. As a result, they have a
      type of growth hormone insensitivity and an inability to grow normally.

      This study is a test to see whether daily dosing with a combination of rhIGF-1 and rhGH will
      help children with IGFD grow taller more quickly than children treated with rhGH alone. The
      study medications, rhIGF-1 and rhGH, are approved by the US Food and Drug Administration
      (FDA) for use in some growth disorders in children, but the combination of rhIGF-1 and rhGH
      in children with IGF-1 deficiency (IGFD) is investigational.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Parents or legally authorized representatives must give signed informed consent before
             any trial-related activities

          -  IGF-1 SDS of ≤ -1 for age and gender

          -  Short stature, as defined by a height SDS of ≤ -2 for age and gender

          -  Chronological age ≥ 5 years

          -  Bone age ≤ 11 years in boys and ≤ 9 years in girls

          -  GH sufficiency, defined as a maximal stimulated GH response of greater than or equal
             to 10 ng/mL at Visit 2 (note: upon approval of the Medical Monitor, the result of a
             prior GH stimulation test may satisfy this requirement).

          -  Prepubertal status

          -  Adequate nutrition as evidenced by a body mass index (BMI) greater than or equal to
             the 5th percentile for age and gender

        Exclusion Criteria:

          -  Severe Primary IGFD (defined as height and IGF-1 SDS ≤ 3, and stimulated GH response
             greater than or equal to 10 ng/mL)

          -  Prior or current use of medications with the potential to alter growth patterns
             including GH, IGF-1, IGFBP-3, gonadotrophin agonists (e.g., Lupron), aromatase
             inhibitors, androgens and estrogens

          -  Known or suspected allergy to rhGH, rhIGF-1 or a constituent of their formulations

          -  Current use of medications for attention deficit disorder

          -  A chronic health condition that requires anti-inflammatory steroids or daily
             medication unless approved by the Medical Monitor