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

NCT01247675 | Completed Phase 2 Results

• 1 other identifier: ACP-001 CT-002
• Study Type: interventional
• Target: 37
• Locations: 4 countries
• Sites: 4

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.

Conditions

Adult Growth Hormone Deficiency

Outcome Measures

Primary Outcomes (2)

• 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.

  Start of study treatment through Week 4

• Incidence of Treatment Emergent Anti-hGH Binding Antibody Formation

  Number of subjects with treatment emergent anti-hGH binding antibodies

  Start of study treatment through Day 42

Secondary Outcomes (2)

• Cmax of hGH

  Days 22 to 29

• Emax of IGF-I

  Days 22 to 29

Study Arms (4)

ACP-001, 0.02 mg hGH/kg/wk

EXPERIMENTAL

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

Drug: ACP-001 (TransCon hGH)

ACP-001, 0.04 mg hGH/kg/wk

EXPERIMENTAL

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

Drug: ACP-001 (TransCon hGH)

ACP-001, 0.08 mg hGH/kg/wk

EXPERIMENTAL

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

Drug: ACP-001 (TransCon hGH)

Omnitrope, 0.04 mg hGH/kg/wk

ACTIVE COMPARATOR

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

Drug: Omnitrope

Interventions

ACP-001 (TransCon hGH) DRUG

s.c., weekly injection

ACP-001, 0.02 mg hGH/kg/wk

Omnitrope DRUG

s.c., daily injection

Omnitrope, 0.04 mg hGH/kg/wk

Eligibility Criteria

• Age: 20 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Ascendis Pharma A/S: lead

Study Sites (4)

Aarhus University Hospital

Aarhus, 8000, Denmark

Location

Charité University Hospital Berlin

Berlin, 12203, Germany

Location

University Hospital Genova

Genova, 16132, Italy

Location

Karolinska University Hospital

Stockholm, 17176, Sweden

Location

MeSH Terms

Conditions

Dwarfism, Pituitary

Interventions

Human Growth Hormone

Condition Hierarchy (Ancestors)

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; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Michael Beckert, MD
• Organization: Ascendis Pharma A/S

mb@ascendispharma.com

(+49) 172-155-2596

Study Officials

• Michael Beckert, MD

  Ascendis Pharma A/S

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 23, 2010
• First Posted: November 24, 2010
• Study Start: November 1, 2010
• Primary Completion: May 1, 2011
• Study Completion: May 1, 2011
• Last Updated: March 9, 2017
• Results First Posted: March 9, 2017

Record last verified: 2017-01

Data Sharing

• IPD Sharing: Will not share

Locations

SE (1); DK (1); IT (1); DE (1)