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A Phase 3 Trial of the Safety, Tolerability and Efficacy of Trans

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

November 22, 2017

  • Clinical Trial Information

    Trial Contact: Tilme, Linda; Parrimon, Yozandra

    Trial Phone: 321.841.5278 ; 321.841.1361

  • IRB No: 16.154.12

    Protocol Abbrev: TransCon hGH CT-301

    Principal Investigator: Joshua H Yang, MD

    Sub Investigators: Garcia, Daniel MD; Faverio, Luis MD; Sabogal, Carlos MD

    Phase: Drug: Phase III

    Age Group: Pediatric

    Secondary Protocol No: TransCon hGH CT-301- Height

    Treatment: Medication

    Applicable Disease Sites: Growth Hormond Deficiency (GHD), Endocrinology

    Therapies Involved: TransCon hGH, Genotropin

    ClinicalTrials.gov ID: NCT02781727

  • Objective

    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).

  • Key Eligibility

    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