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

Ascendis Pharma

Status and phase: Completed; Phase 2

Conditions: Growth Hormone Deficiency (GHD)

Treatments: Drug: Human Growth Hormone; Drug: ACP-001

Study type: Interventional

Funder types: Industry

Identifiers

NCT01947907

ACP-001_CT-004

Details and patient eligibility

About

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.

Enrollment: 53 patients

Sex: All

Ages: 3 to 12 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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53 participants in 4 patient groups

ACP-001, dose-level 1

Experimental group

Description:

Once weekly subcutaneous injection of ACP-001

Treatment:

Drug: ACP-001

ACP-001, dose-level 2

Experimental group

Description:

Once weekly subcutaneous injection of ACP-001

Treatment:

Drug: ACP-001

ACP-001, dose-level 3

Experimental group

Description:

Once weekly subcutaneous injection of ACP-001

Treatment:

Drug: ACP-001

Human Growth Hormone

Active Comparator group

Description:

Once daily subcutaneous injection of human Growth Hormone (rhGH)

Treatment:

Drug: Human Growth Hormone