Safety and Efficacy Phase 2 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children

Inclusion Criteria

• Pre-pubertal child aged ≥ 3 yrs old and not above 10 years for girls or 11 years for boys with either isolated GHD, or GH insufficiency as part of multiple pituitary hormone deficiency.
• Confirmed diagnosis of GHD by two different GH provocation tests defined as a peak plasma GH level of ≤7 ng/ml, determined by central laboratory using a validated assay .
• Bone age (BA) is not older than chronological age and should be no greater than 9 years for girls and 10 years for boys.
• Without prior exposure to any r-hGH therapy.
• Normal birth size, birth weight and length for gestational age
• Impaired height and height velocity defined as:
• Height (HT) of at least 2.0 standard deviations (SD) below the mean height for chronological age (CA) and gender according to the standards from Prader et. al, 1989 , (HT SDS ≤ -2.0).
• Annualized height velocity (HV) below the 25th percentile for CA (HV 110 mg/dl or 6.1 mmol/l) after repeated blood analysis.
• Chromosomal abnormalities and medical "syndromes" (Turner's syndrome, Laron syndrome, Noonan syndrome, Prader-Willi Syndrome, Russell-Silver Syndrome, SHOX mutations/deletions and skeletal dysplasias), with the exception of septo-optic dysplasia.
• Closed epiphyses.
• Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids and methylphenidate for attention deficit hyperactivity disorder (ADHD), with the exception of hormone replacement therapies (thyroxine, hydrocortisone, desmopressin (DDAVP))
• Children requiring glucocorticoid therapy (e.g. asthma) who are taking a dose of greater than 400 µg/d of inhaled budesonide or equivalents for longer than 1 month during a calendar year.
• Major medical conditions and/or presence of contraindication to r-hGH treatment.
• Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis.
• Drug, substance, or alcohol abuse.
• Known hypersensitivity to the components of study medication.
• Other causes of short stature such as coeliac disease, hypothyroidism and rickets.
• The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct.
• Participation in any other trial of an investigational agent within 30 days prior to Screening.