Adult Growth Hormone DeficiencyPhase 2NCT01247675
A Safety, Pharmacokinetic and Pharmacodynamic Study of ACP-001 (TransCon hGH) in Adults With Growth Hormone Deficiency
A Phase 2 study testing ACP-001 (TransCon hGH) and Omnitrope in people with Adult Growth Hormone Deficiency, run by Ascendis Pharma A/S at 4 sites in 4 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 20 to 70 years
- Conditions the study is for
- Adult Growth Hormone Deficiency
- What is being tested?
- ACP-001 (TransCon hGH) and Omnitrope
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 37 participants
- Where
- 4 sitesin 4 countries
- Study dates
- Nov 2010to May 2011
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
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- 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
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
About this study
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
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
- Being tested
- ACP-001 (TransCon hGH), Omnitrope
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ACP-001 (TransCon hGH)Drug
s.c., weekly injection
ACP-001 (TransCon hGH)Drug
s.c., weekly injection
ACP-001 (TransCon hGH)Drug
s.c., weekly injection
OmnitropeDrug
s.c., daily injection
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: 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 Receives: 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 Receives: Drug: Omnitrope |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: Start of study treatment through Day 42
Secondary outcomes (2)
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
Measured: Days 22 to 29
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
Measured: Days 22 to 29
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2010 (estimated)
- Study startNov 2010
- Primary completionMay 2011
- Study completionMay 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Adult Growth Hormone DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ascendis Pharma A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, Ascendis Pharma A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01247675
- Sponsor's protocol ID
- ACP-001 CT-002
- Status last verified
- Jun 2026
- First submitted
- 23 Nov 2010
- Last updated
- 8 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No