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

Primary Purpose

Adult Growth Hormone Deficiency

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

ACP-001 (TransCon hGH)

Omnitrope

About this trial

This is an interventional treatment trial for Adult Growth Hormone Deficiency

Eligibility Criteria

20 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Aarhus University Hospital
Charité University Hospital Berlin
University Hospital Genova
Karolinska University Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Active Comparator

Arm Label

ACP-001, 0.02 mg hGH/kg/wk

ACP-001, 0.04 mg hGH/kg/wk

ACP-001, 0.08 mg hGH/kg/wk

Omnitrope, 0.04 mg hGH/kg/wk

Arm Description

Once weekly subcutaneous injection of ACP-001 equivalent to 0.02 mg hGH/kg/week for 4 weeks

Once weekly subcutaneous injection of ACP-001 equivalent to 0.04 mg hGH/kg/week for 4 weeks

Once weekly subcutaneous injection of ACP-001 equivalent to 0.08 mg hGH/kg/week for 4 weeks

Once daily subcutaneous injection of human Growth Hormone (Omnitrope) equivalent to 0.04 mg hGH/kg/week for 4 weeks

Outcomes

Primary Outcome Measures

Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)

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

Number of subjects with treatment emergent anti-hGH binding antibodies

Secondary Outcome Measures

Cmax of hGH

As part of the following endpoint: Pharmacokinetic (PK) profile of serum human Growth Hormone (hGH) from ACP-001 treated dose groups compared to the PK profile of hGH from the daily Omnitrope treated group. Cmax (maximum value of concentration) values at Week 4

Emax of IGF-I

As part of the following endpoint: Pharmacodynamic (PD) response of serum Insulin-like Growth Factor-I (IGF-I) from ACP-001 treated dose groups compared to the PD response of IGF-I from the daily Omnitrope treated group. Emax (maximum observed response) values at Week 4

Full Information

NCT ID

NCT01247675

First Posted

November 23, 2010

Last Updated

January 20, 2017

Sponsor

Ascendis Pharma A/S

1. Study Identification

Unique Protocol Identification Number

NCT01247675

Brief Title

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

2. Study Status

Record Verification Date

January 2017

Overall Recruitment Status

Completed

Study Start Date

November 2010 (undefined)

Primary Completion Date

May 2011 (Actual)

Study Completion Date

May 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Ascendis Pharma A/S

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Adult Growth Hormone Deficiency

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

37 (Actual)

8. Arms, Groups, and Interventions

Arm Title

ACP-001, 0.02 mg hGH/kg/wk

Arm Type

Experimental

Arm Description

Once weekly subcutaneous injection of ACP-001 equivalent to 0.02 mg hGH/kg/week for 4 weeks

Arm Title

ACP-001, 0.04 mg hGH/kg/wk

Arm Type

Experimental

Arm Description

Once weekly subcutaneous injection of ACP-001 equivalent to 0.04 mg hGH/kg/week for 4 weeks

Arm Title

ACP-001, 0.08 mg hGH/kg/wk

Arm Type

Experimental

Arm Description

Once weekly subcutaneous injection of ACP-001 equivalent to 0.08 mg hGH/kg/week for 4 weeks

Arm Title

Omnitrope, 0.04 mg hGH/kg/wk

Arm Type

Active Comparator

Arm Description

Once daily subcutaneous injection of human Growth Hormone (Omnitrope) equivalent to 0.04 mg hGH/kg/week for 4 weeks

Intervention Type

Drug

Intervention Name(s)

ACP-001 (TransCon hGH)

Intervention Description

s.c., weekly injection

Intervention Type

Drug

Intervention Name(s)

ACP-001 (TransCon hGH)

Intervention Description

s.c., weekly injection

Intervention Type

Drug

Intervention Name(s)

ACP-001 (TransCon hGH)

Intervention Description

s.c., weekly injection

Intervention Type

Drug

Intervention Name(s)

Omnitrope

Intervention Description

s.c., daily injection

Primary Outcome Measure Information:

Title

Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)

Description

Time Frame

Start of study treatment through Week 4

Title

Incidence of Treatment Emergent Anti-hGH Binding Antibody Formation

Description

Number of subjects with treatment emergent anti-hGH binding antibodies

Time Frame

Start of study treatment through Day 42

Secondary Outcome Measure Information:

Title

Cmax of hGH

Description

Time Frame

Days 22 to 29

Title

Emax of IGF-I

Description

Time Frame

Days 22 to 29

10. Eligibility

Sex

All

Minimum Age & Unit of Time

20 Years

Maximum Age & Unit of Time

70 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Michael Beckert, MD

Organizational Affiliation

Ascendis Pharma A/S

Official's Role

Study Director

Facility Information:

Facility Name

Aarhus University Hospital

City

Aarhus

ZIP/Postal Code

8000

Country

Denmark

Facility Name

Charité University Hospital Berlin

City

Berlin

ZIP/Postal Code

12203

Country

Germany

Facility Name

University Hospital Genova

City

Genova

ZIP/Postal Code

16132

Country

Italy

Facility Name

Karolinska University Hospital

City

Stockholm

ZIP/Postal Code

17176

Country

Sweden

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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