Growth Hormone Deficiency (GHD)Phase 2NCT01947907
Safety, PK/PD (Pharmacokinetics/Pharmacodynamics) and Efficacy of ACP-001 Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)
A Phase 2 study testing ACP-001 and Human Growth Hormone in people with Growth Hormone Deficiency (GHD), run by Ascendis Pharma A/S at 36 sites in 14 countries.
Trial summary
A six month study of ACP-001, a long-acting growth hormone product, versus standard human growth hormone therapy. ACP-001 will be given once-a-week, standard human growth hormone (hGH) will be given on a daily basis. Read more about this study
- Who is it for?
- Ages 3 to 12 years
- Conditions the study is for
- Growth Hormone Deficiency (GHD)
- What is being tested?
- ACP-001 and Human Growth Hormone
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 53 participants
- Where
- 36 sitesin 14 countries
- Study dates
- Jul 2013to Sep 2015
- 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 3 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Prepubertal children, Tanner stage 1
- Diagnosis of GHD, confirmed by two stimulation tests
- Bone age not greater than chronological age
- Impaired height and height velocity
- BMI within +/- 2 SD (standard deviations)
- Baseline IGF-1 (insulin-like growth factor)
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- Normal fundoscopy
- Stable hormonal replacement therapy (other than hGH)
- Written Informed Consent
Who cannot join
- Prior exposure to rhGH or IGF-I
- Past or present intracranial tumor; history or presence of malignant disease
- Small for gestational age (SGA)
- Malnutrition
- Psychosocial dwarfism
- Coeliac disease
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- Anti-hGH antibodies
- Diabetes mellitus
- Chromosomal abnormalities (e.g. Turner syndrome, SHOX)
- Closed epiphyses
- Known or suspected HIV infection
Trial locations and contacts
36 sites in 14 countries.
36 sites in 14 countries
Ukraine5 sites
Donetsk Regional Children Clinical Hospital
Donetsk
Kharkiv National Medical University
Kharkiv
Institute of Endocrinology and Metabolism
Kiev
Ukrainian Children Specialized Clinical Hospital
Kiev
Odessa National Medical University
Odesa
Egypt4 sites
El Shatby University Hospital
Alexandria
Ain Shams University Hospital
Cairo
Cairo University Hospital
Cairo
El Mansoura University Hospital
El Mansoura
Hungary4 sites
Buda Children's Hospital
Budapest
Heim Pal Children's Hospital
Budapest
University of Pecs
Pécs
University of Szeged
Szeged
Poland4 sites
University Medical Hospital
Katowice
Medical University of Lublin
Lublin
Regional Hospital N°2 Rzeszow
Rzeszów
Children's Memorial Health Institute Warsaw
Warsaw
Russia4 sites
Federal State Budgetary Institution
Moscow
St. Petersburg State Pediatric Medical Academy
Saint Petersburg
Samara State Medical University
Samara
Bashkir State Medical University
Ufa
France3 sites
Hôpital des enfants Pellegrin
Bordeaux
Hôpital Jeanne de Flandre
Lille
Hôpital Femme-Mère-Enfant
Lyon
Romania3 sites
Emergency Clinical Hospital Cluj-Napoca
Cluj-Napoca
St. Spiridon County Clinic Emergency Hospital
Iași
Louis Turcanu Emergency Hospital for Children Timisoara
Timișoara
Germany2 sites
University Hospital Leipzig
Leipzig
University Children's Hospital Magdeburg
Magdeburg
Turkey (Türkiye)2 sites
Ankara University School of Medicine
Ankara
Ege Üniversity
Izmir
Bulgaria1 site
University Multiprofile Hospital for Active Treatment (UMHAT) "Sv. Marina"
Varna
About this study
Official title: A Multicenter, Phase 2, Randomized, Open Label, Active-controlled, Parallel-group Study Investigating the Safety, Tolerability, and Efficacy of Different Dose Levels of ACP-001 Administered Once Weekly Versus Standard Daily rhGH Replacement Therapy in Pre-pubertal Children With Growth Hormone Deficiency (GHD)
A six month study of ACP-001, a long-acting growth hormone product, versus standard human growth hormone therapy. ACP-001 will be given once-a-week, standard human growth hormone (hGH) will be given on a daily basis. The primary aim is to demonstrate safety, pharmacokinetics and pharmacodynamics over a period of six months. A secondary objective is the comparison of height velocity (HV) of the ACP-001 treated groups to the daily hGH treatment group.
- Conditions
- Growth Hormone Deficiency (GHD)
- Being tested
- ACP-001, Human Growth Hormone
- Keywords
- Human Growth Hormone, rhGH, hGH, GHD
- 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-001Drug
Once weekly subcutaneous injection
Human Growth HormoneDrug
Also called Somatropin
Once daily subcutaneous injection of human Growth Hormone
| Study group | Role | What they receive |
|---|---|---|
| ACP-001, dose-level 1 | Experimental | Once weekly subcutaneous injection of ACP-001 Receives: Drug: ACP-001 |
| ACP-001, dose-level 2 | Experimental | Once weekly subcutaneous injection of ACP-001 Receives: Drug: ACP-001 |
| ACP-001, dose-level 3 | Experimental | Once weekly subcutaneous injection of ACP-001 Receives: Drug: ACP-001 |
| Human Growth Hormone | Active comparator | Once daily subcutaneous injection of human Growth Hormone (rhGH) Receives: Drug: Human Growth Hormone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Anti-hGH Binding Antibody Formation
Number of subjects with positive results for anti-hGH binding antibodies at two consecutive post-dose visits
Measured: Visit 2 - Visit 5
Incidence of Anti-hGH Neutralizing Antibody Formation
Number of subjects with positive results for anti-hGH neutralizing antibodies at two consecutive post-dose visits
Measured: Visit 2 - Visit 5
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 anamnestic data and records in the Patient Diary. Assessments included pain, redness, bruising, swelling, and itching. Every subject was counted only once within each symptom category.
Measured: Start of study treatment through Visit 5 (Week 27)
Cmax of hGH
As part of the following endpoint:
PK profile of serum hGH from ACP-001 treated patients compared between ACP-001 dose groups and to the pharmacokinetic (PK) profile of hGH from the daily rhGH groups during visit 1 and 3.
Uncorrected Cmax (maximum value of concentration) values at Visit 3 (Week 13)
Measured: 0 hours to 168 hours at Visit 3 (Week 13)
AUC0-168h of hGH
As part of the following endpoint:
PK profile of serum hGH from ACP-001 treated patients compared between ACP-001 dose groups and to the PK profile of hGH from the daily rhGH group during Visit 1 and Visit 3
Uncorrected AUC0-168h (area under the curve from 0h to 168h) values at Visit 3 (Week 13)
Measured: 0 hours to 168 hours at Visit 3 (Week 13)
E-Trough of IGF-1
As part of the following endpoint:
PD profile of serum IGF-1 during Visit 1 and Visit 3 compared between the ACP-001 dose groups and to the daily rhGH group
Uncorrected E-Trough (the pre-dose efficacy response) values at Week 13
Measured: 0 hours to 168 hours at Visit 3 (Week 13)
Emax of IGF-1
As part of the following endpoint:
PD profile of serum IGF-1 during V1 and V3 compared between the ACP-001 dose groups and to the daily rhGH group
Measured: 0 hours to 168 hours at Visit 3 (Week 13)
AUEC0-168h of IGF-1
As part of the following endpoint:
PD profile of serum IGF-1 during V1 and V3 compared between the ACP-001 dose groups and to the rhGH group.
Uncorrected AUEC0-168 (area under the efficacy curve from 0h-168h) values at Week 13
Measured: 0 hours to 168 hours at Visit 3 (Week 13)
Secondary outcomes (1)
Annualized Height Velocity
Annualized HV during treatment with ACP-001 or daily rhGH at the end of 6 months, for each ACP-001 dose group and for the daily rhGH dose group
Measured: Baseline to 6 months (Visit 5)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2013 (estimated)
- Study startJul 2013
- Primary completionJul 2015
- Study completionSep 2015
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Growth Hormone Deficiency (GHD)Studies 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
- 53 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
- NCT01947907
- Sponsor's protocol ID
- ACP-001_CT-004
- Status last verified
- Jun 2026
- First submitted
- 3 Jun 2013
- Last updated
- 8 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Chatelain P, Malievskiy O, Radziuk K, Senatorova G, Abdou MO, Vlachopapadopoulou E, Skorodok Y, Peterkova V, Leff JA, Beckert M; TransCon GH Working Group. A Randomized Phase 2 Study of Long-Acting TransCon GH vs Daily GH in Childhood GH Deficiency. J Clin Endocrinol Metab. 2017 May 1;102(5):1673-1682. doi: 10.1210/jc.2016-3776.
PubMed 28201598