A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)

Launched by ASCENDIS PHARMA ENDOCRINOLOGY DIVISION A/S · May 20, 2016

Keywords

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Gender

ALL

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