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

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