A Safety, Pharmacokinetic and Pharmacodynamic Study of ACP-001 (TransCon PEG hGH) in Adults With Growth Hormone Deficiency

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S
ClinicalTrials.gov Identifier:
NCT01247675
First received: November 23, 2010
Last updated: June 2, 2013
Last verified: June 2013
  Purpose

This study investigates the pharmacokinetic profile (PK) and pharmacodynamic response (PD) of three different doses of ACP-001 given once-a-week compared to one dose-level of an approved daily human growth hormone product over a period of 4 weeks (4 weekly administrations versus 28 daily administrations).


Condition Intervention Phase
Growth Hormone Deficient Adults
Drug: ACP-001 (TransCon PEG hGH)
Drug: Human Growth Hormone
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Pharmacokinetics/Dynamics Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official 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

Resource links provided by NLM:


Further study details as provided by Ascendis Pharma A/S:

Primary Outcome Measures:
  • Safety and Tolerability (Adverse events, vital signs, safety laboratory, immunogenicity) [ Time Frame: 4 weeks ] [ Designated as safety issue: Yes ]
    Adverse events, vital signs, safety laboratory, immunogenicity


Secondary Outcome Measures:
  • Change of IGF-I levels over time [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]
    Change from baseline after wash-out phase

  • Change of IGFBP-3 over time [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]
    Change from baseline after wash-out phase


Enrollment: 37
Study Start Date: November 2010
Study Completion Date: May 2011
Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ACP-001, dose-level 1 Drug: ACP-001 (TransCon PEG hGH)
s.c., weekly injection
Experimental: ACP-001, dose-level 2 Drug: ACP-001 (TransCon PEG hGH)
s.c., weekly injection
Experimental: ACP-001, dose-level 3 Drug: ACP-001 (TransCon PEG hGH)
s.c., weekly injection
Active Comparator: Daily Human Growth Hormone Drug: Human Growth Hormone
s.c., daily injection over 28 days

  Eligibility

Ages Eligible for Study:   20 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female between 20 to 70 years
  • Body Mass Index (BMI, kg/m2) of 19.0 to 36.0 kg/m2, both inclusive
  • 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 (Consensus Guidelines 1998 and 2007)
  • Fertile females must agree to use appropriate contraceptive methods and have a negative pregnancy test at inclusion
  • GH replacement therapy for at least 3 months
  • Willing to maintain current activity level during the trial
  • Subjects are able and willing to provide written informed consent and authorization for protected health information disclosure in accordance with Good Clinical Practice (GCP)

Exclusion Criteria:

  • History of hypersensitivity and/or idiosyncrasy to any of the test compounds or excipients employed in this study.
  • 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.
  • Active malignant disease or malignant disease within the last 5 years
  • Proliferative retinopathy judged by retina-photo within the last year
  • Heart insufficiency as judged by the investigator and/or NYHA 3 or greater (NYHA criteria for diagnosis of diseases of the heart, 1994)
  • Subjects with uncontrolled diabetes with an HbA1c above 8.0% and/or insulin treatment
  • Stable pituitary hormone replacement therapy for less than 3 months
  • Impaired liver function as judged by the investigator or hepatic transaminases > 2 times the upper limit of normal
  • Impaired kidney function as judged by the investigator and/or creatinine clearance <50 mL/min and/or serum creatinine > 1.4 mg/dL
  • Participation in another interventional clinical study involving an investigational compound within 3 months prior to enrolment in this study or participation in another interventional clinical study involving an investigational compound during this study.
  • 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.
  • History or presence of alcohol abuse or drug abuse.
  • Patients with known history for, or presence of, anti-hGH and / or anti-PEG antibodies
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01247675

Locations
Denmark
Aarhus University Hospital
Aarhus, Denmark, 8000
Germany
Charité University Hospital Berlin
Berlin, Germany, 12203
Italy
University Hospital Genova
Genova, Italy, 16132
Sweden
Karolinska University Hospital
Stockholm, Sweden, 17176
Sponsors and Collaborators
Ascendis Pharma A/S
Investigators
Study Director: Michael Beckert, MD Ascendis Pharma A/S
  More Information

No publications provided

Responsible Party: Ascendis Pharma A/S
ClinicalTrials.gov Identifier: NCT01247675     History of Changes
Other Study ID Numbers: ACP-001 CT002
Study First Received: November 23, 2010
Last Updated: June 2, 2013
Health Authority: Austria: Agency for Health and Food Safety
Denmark: Danish Medicines Agency
Germany: Federal Institute for Drugs and Medical Devices
Italy: The Italian Medicines Agency
Sweden: Medical Products Agency

Additional relevant MeSH terms:
Dwarfism, Pituitary
Endocrine System Diseases
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 20, 2014