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
Overview
- Phase
- Phase 2
- Intervention
- ACP-001 (TransCon hGH)
- Conditions
- Adult Growth Hormone Deficiency
- Sponsor
- Ascendis Pharma A/S
- Enrollment
- 37
- Locations
- 4
- Primary Endpoint
- Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)
- Status
- Completed
- Last Updated
- 9 years ago
Overview
Brief Summary
This study investigates the safety, tolerability, 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) in adults with Growth Hormone Deficiency.
Investigators
Eligibility 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.
Arms & Interventions
ACP-001, 0.02 mg hGH/kg/wk
Once weekly subcutaneous injection of ACP-001 equivalent to 0.02 mg hGH/kg/week for 4 weeks
Intervention: ACP-001 (TransCon hGH)
ACP-001, 0.04 mg hGH/kg/wk
Once weekly subcutaneous injection of ACP-001 equivalent to 0.04 mg hGH/kg/week for 4 weeks
Intervention: ACP-001 (TransCon hGH)
ACP-001, 0.08 mg hGH/kg/wk
Once weekly subcutaneous injection of ACP-001 equivalent to 0.08 mg hGH/kg/week for 4 weeks
Intervention: ACP-001 (TransCon hGH)
Omnitrope, 0.04 mg hGH/kg/wk
Once daily subcutaneous injection of human Growth Hormone (Omnitrope) equivalent to 0.04 mg hGH/kg/week for 4 weeks
Intervention: Omnitrope
Outcomes
Primary Outcomes
Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)
Time Frame: Start of study treatment through Week 4
Assessment of local tolerability was performed by examining injection sites by the investigator during study visits, and on the basis of records in the Patient Diary. Assessments included erythema, swelling, or pain.
Incidence of Treatment Emergent Anti-hGH Binding Antibody Formation
Time Frame: Start of study treatment through Day 42
Number of subjects with treatment emergent anti-hGH binding antibodies
Secondary Outcomes
- Cmax of hGH(Days 22 to 29)
- Emax of IGF-I(Days 22 to 29)