Safety, PK/PD (Pharmacokinetics/Pharmacodynamics) and Efficacy of ACP-001 Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)

A six month study of ACP-001, a long-acting growth hormone product, versus standard human growth hormone therapy. ACP-001 will be given once-a-week, standard human growth hormone (hGH) will be given on a daily basis. The primary aim is to demonstrate safety, pharmacokinetics and pharmacodynamics over a period of six months. A secondary objective is the comparison of height velocity (HV) of the ACP-001 treated groups to the daily hGH treatment group.

Inclusion Criteria: * Prepubertal children, Tanner stage 1 * Diagnosis of GHD, confirmed by two stimulation tests * Bone age not greater than chronological age * Impaired height and height velocity * BMI within +/- 2 SD (standard deviations) * Baseline IGF-1 (insulin-like growth factor) * Normal fundoscopy * Stable hormonal replacement therapy (other than hGH) * Written Informed Consent Exclusion Criteria: * Prior exposure to rhGH or IGF-I * Past or present intracranial tumor; history or presence of malignant disease * Small for gestational age (SGA) * Malnutrition * Psychosocial dwarfism * Coeliac disease * Anti-hGH antibodies * Diabetes mellitus * Chromosomal abnormalities (e.g. Turner syndrome, SHOX) * Closed epiphyses * Known or suspected HIV infection