Clinical trial NCT01247675: A Safety, Pharmacokinetic and Pharmacodynamic Study of ACP-001 (TransCon hGH) in Adults With Growth Hormone Deficiency
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Adult Growth Hormone Deficiency
Lead sponsor: Ascendis Pharma A/S
Sponsor class: INDUSTRY
First posted: 2010-11-24
Enrollment: 37
Sex: ALL
Minimum age (years): 20
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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.
* 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

Primary outcomes:
measure: Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)
timeFrame: Start of study treatment through Week 4
measure: Incidence of Treatment Emergent Anti-hGH Binding Antibody Formation
timeFrame: Start of study treatment through Day 42
Secondary outcomes:
measure: Cmax of hGH
timeFrame: Days 22 to 29
measure: Emax of IGF-I
timeFrame: Days 22 to 29
Other outcomes:
Locations:
city: Aarhus
country: Denmark
facility: Aarhus University Hospital
city: Berlin
country: Germany
facility: Charité University Hospital Berlin
city: Genova
country: Italy
facility: University Hospital Genova
city: Stockholm
country: Sweden
facility: Karolinska University Hospital
Study officials:
affiliation: Ascendis Pharma A/S
name: Michael Beckert, MD
role: STUDY_DIRECTOR
References: