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

• Status: Completed
• Trial end date: 2015-09-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ascendis Pharma A/S
• Treatments: Hormones

Criteria

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