A Phase 2, Multiple Dose, Open-Label, Parallel-Group, Active Controlled, Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of ACP-001 in Adult Patients with Growth Hormone Deficiency (AGHD)

• Register: EUCTR
• Last refreshed on: 19 November 2012
• Main ID: EUCTR2010-021523-28-AT
• Date of registration: 20/07/2010
• Prospective Registration: Yes
• Primary sponsor: Ascendis Pharma A/S
• Public title: A Phase 2, Multiple Dose, Open-Label, Parallel-Group, Active Controlled, Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of ACP-001 in Adult Patients with Growth Hormone Deficiency (AGHD)
• Scientific title: A Phase 2, Multiple Dose, Open-Label, Parallel-Group, Active Controlled, Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of ACP-001 in Adult Patients with Growth Hormone Deficiency (AGHD)
• Date of first enrolment: 02/09/2010
• Target sample size: 36
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-021523-28
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: yes Other trial design description: partly laboratory blinded If controlled, specify comparator, Other Medicinial Product: yes Placebo: no Other: no

Countries of recruitment

Austria, Denmark, Germany, Italy, Sweden

Key inclusion & exclusion criteria

Inclusion criteria:
1. Male or female between 20 to 70 years
2. Body Mass Index (BMI, kg/m2) of 19.0 to 36.0 kg/m2, both inclusive
3. Adult Growth Hormone Deficient (AHGD) patients with documented growth hormone deficiency as defined in the Consensus guidelines for the diagnosis and treatment of adults with GH deficiency II (Ho, 2007)
4. Fertile females must agree to use appropriate contraceptive methods and have a negative pregnancy test at inclusion
5. GH replacement therapy for at least 3 months
6. Willing to maintain current activity level during the trial
7. Subjects are able and willing to provide written informed consent and authorization for protected health information disclosure in accordance with Good Clinical Practice (GCP

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. History of hypersensitivity and/or idiosyncrasy to any of the test compounds or excipients employed in this study.
2. Females of childbearing potential who are pregnant, breast-feeding or intend to become pregnant or are not using adequate contraceptive methods. Reliable methods for women are orally administered hormonal contraceptives, surgical intervention (e.g. tubal ligation), intrauterine device (IUD) and sexual abstinence.
3. Active malignant disease or malignant disease within the last 5 years
4. Proliferative retinopathy judged by retina-photo within the last year
5. Heart insufficiency as judged by the investigator and/or NYHA 3 or greater
6. Subjects with uncontrolled diabetes with an HbA1c above 8.0% and/or insulin treatment
7. Stable pituitary replacement therapy for less than 3 months
8. Impaired liver function as judged by the investigator or h (hepatic transaminases > 2 times the upper limit of normal
9. Impaired kidney function as judged by the investigator and/or (creatinine clearance <50 ml/min and/or serum creatinine > 1,4 mg/dL
10. Participation in another interventional clinical study involving an investigational compound within 3 months prior to enrolment in this study or during the study.
11. Subjects who are unable to comply with the requirements of the study or who in the opinion of the investigator should not participate in the study.
12. History or presence of alcohol abuse or drug abuse.
13. Patients with known history for, or presence of, anti-hGH and / or anti-PEG antibodies


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Growth Hormone Deficiency
MedDRA version: 12.1 Level: PT Classification code 10056438 Term: Growth hormone deficiency

Intervention(s)

Product Name: ACP-001
Product Code: ACP-001
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: SOMATROPIN
CAS Number: 12629-01-5
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 32-

Trade Name: Omnitrope®
Pharmaceutical Form: Solution for injection
INN or Proposed INN: SOMATROPIN
CAS Number: 12629-01-5
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 3.3-

Primary Outcome(s)

Secondary Objective: To assess the pharmacokinetics of three different dose-levels of ACP-001 over a 4-week course of treatment

To assess the pharmacodynamic response of three different dose-levels of ACP-001 and the active comparator, daily Human Growth Hormone, over a 4-week course of treatment

Primary end point(s): Pharmacokinetics (PK):
• Change in TransCon PEG hGH serum concentration from baseline to last treatment period
• Change in hGH serum concentration from baseline to last treatment period

Efficacy/Pharmacodynamics (PD):
• Primary efficacy: The mean absolute and percent change of plasma IGF-I from baseline to last treatment period
• Secondary efficacy: The mean absolute and percent changes in plasma IGFBP-3 from baseline to last treatment period; alterations in GHBP from baseline to last treatment period

Safety and Tolerability:
Adverse events, injection site reactions, physical examinations, laboratory measurements, with particular emphasis on changes in alanine aminotransferase (ALT), aspartate aminotransferase (AST), blood glucose, immunogenicity (anti-hGH antibodies; anti-PEG antibodies) and injection site reactions.

Main Objective: The primary objective of the study is to assess the safety and tolerability of ACP-001 under multiple-dose conditions in adults patients with Growth Hormone Deficiency

Secondary ID(s)

ACP-001 CT-002