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CompletedNCT01947907Updated Jul 8, 2026Results posted

Safety, PK/PD (Pharmacokinetics/Pharmacodynamics) and Efficacy of ACP-001 Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)

A Phase 2 interventional study of ACP-001 and Human Growth Hormone in Growth Hormone Deficiency (GHD), sponsored by Ascendis Pharma A/S. Completed at 36 sites in 14 countries. Open to participants aged 3 Years to 12 Years. Per ClinicalTrials.gov, last updated 2026-07-08.

Sponsored by Ascendis Pharma A/S · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
53
Allocation
Randomized
Ages
3 Years to 12 Years
Sex
All
01

Study 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. 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.

02

Conditions studied

  • Growth Hormone Deficiency (GHD)

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Keywords

  • Human Growth Hormone
  • rhGH
  • hGH
  • GHD
03

Who can participate

Ages eligible
3 Years to 12 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • 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)
  • Normal fundoscopy
  • Stable hormonal replacement therapy (other than hGH)
  • Written Informed Consent

Exclusion criteria

Exclusion Criteria:

  • 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
  • Anti-hGH antibodies
  • Diabetes mellitus
  • Chromosomal abnormalities (e.g. Turner syndrome, SHOX)
  • Closed epiphyses
  • Known or suspected HIV infection
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
53 participants (actual)

Study arms

  • Experimental
    ACP-001, dose-level 1

    Once weekly subcutaneous injection of ACP-001

    Drug: ACP-001

  • Experimental
    ACP-001, dose-level 2

    Once weekly subcutaneous injection of ACP-001

    Drug: ACP-001

  • Experimental
    ACP-001, dose-level 3

    Once weekly subcutaneous injection of ACP-001

    Drug: ACP-001

  • Active comparator
    Human Growth Hormone

    Once daily subcutaneous injection of human Growth Hormone (rhGH)

    Drug: Human Growth Hormone

Interventions

  • DrugACP-001

    Once weekly subcutaneous injection

  • DrugHuman Growth Hormone

    Once daily subcutaneous injection of human Growth Hormone

    Also known as: Somatropin

05

What researchers measure

Primary outcomes

  1. Incidence of Anti-hGH Binding Antibody Formation

    Number of subjects with positive results for anti-hGH binding antibodies at two consecutive post-dose visits

    Time frame: Visit 2 - Visit 5

  2. Incidence of Anti-hGH Neutralizing Antibody Formation

    Number of subjects with positive results for anti-hGH neutralizing antibodies at two consecutive post-dose visits

    Time frame: Visit 2 - Visit 5

  3. 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.

    Time frame: Start of study treatment through Visit 5 (Week 27)

  4. 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)

    Time frame: 0 hours to 168 hours at Visit 3 (Week 13)

  5. 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)

    Time frame: 0 hours to 168 hours at Visit 3 (Week 13)

  6. 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

    Time frame: 0 hours to 168 hours at Visit 3 (Week 13)

  7. 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

    Time frame: 0 hours to 168 hours at Visit 3 (Week 13)

  8. 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

    Time frame: 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

    Time frame: Baseline to 6 months (Visit 5)

06

Results

Posted Jan 19, 2017

Participant flow

Participant flow — Overall Study
MilestoneACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth Hormone
Started12141413
Completed12141413
Not completed0000

Outcome measures

PrimaryIncidence of Anti-hGH Binding Antibody Formation

Number of subjects with positive results for anti-hGH binding antibodies at two consecutive post-dose visits

Time frame:
Visit 2 - Visit 5
Reported as:
Number · participants
Incidence of Anti-hGH Binding Antibody Formation
participantsACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth Hormone
Incidence of Anti-hGH Binding Antibody Formation1000
PrimaryIncidence of Anti-hGH Neutralizing Antibody Formation

Number of subjects with positive results for anti-hGH neutralizing antibodies at two consecutive post-dose visits

Time frame:
Visit 2 - Visit 5
Reported as:
Number · participants
Incidence of Anti-hGH Neutralizing Antibody Formation
participantsACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth Hormone
Incidence of Anti-hGH Neutralizing Antibody Formation0000
PrimaryNumber 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.

Time frame:
Start of study treatment through Visit 5 (Week 27)
Reported as:
Number · Number of subjects with any symptom
Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)
Number of subjects with any symptomACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth Hormone
Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator)7666
PrimaryCmax 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)

Time frame:
0 hours to 168 hours at Visit 3 (Week 13)
Reported as:
Mean · ng/mL
Cmax of hGH
ng/mLACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth Hormone
Cmax of hGH12.558 ± 8.67813.418 ± 9.42831.8 ± 17.49916.612 ± 12.777
PrimaryAUC0-168h 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)

Time frame:
0 hours to 168 hours at Visit 3 (Week 13)
Reported as:
Mean · h*ng/mL
AUC0-168h of hGH
h*ng/mLACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth Hormone
AUC0-168h of hGH696.34 ± 410.096787.41 ± 483.1692167.43 ± 1064.729556.88 ± 412.618
PrimaryE-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

Time frame:
0 hours to 168 hours at Visit 3 (Week 13)
Reported as:
Mean · ng/mL
E-Trough of IGF-1
ng/mLACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3
E-Trough of IGF-197.17 ± 50.53156 ± 76.235167.83 ± 74.01
PrimaryEmax 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

Time frame:
0 hours to 168 hours at Visit 3 (Week 13)
Reported as:
Mean · ng/mL
Emax of IGF-1
ng/mLACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3
Emax of IGF-1209.5 ± 120.189276 ± 126.632289.92 ± 129.469
PrimaryAUEC0-168h 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

Time frame:
0 hours to 168 hours at Visit 3 (Week 13)
Reported as:
Mean · h*ng/mL
AUEC0-168h of IGF-1
h*ng/mLACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3
AUEC0-168h of IGF-128526.19 ± 15756.4435591.94 ± 17068.5936066.01 ± 17379.44
SecondaryAnnualized 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

Time frame:
Baseline to 6 months (Visit 5)
Reported as:
Mean · cm/year
Annualized Height Velocity
cm/yearACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth Hormone
Annualized Height Velocity11.93 ± 4.06612.89 ± 3.46413.85 ± 4.00911.64 ± 3.592

Adverse events

Collected over From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events). Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
ACP-001, Dose-level 1—1/12 (8.3%)7/12 (58.3%)
ACP-001, Dose-level 2—0/14 (0%)6/14 (42.9%)
ACP-001, Dose-level 3—0/14 (0%)8/14 (57.1%)
Human Growth Hormone—0/13 (0%)8/13 (61.5%)
Most frequent serious events
Most frequent serious events
EventACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth Hormone
Inguinal herniaGastrointestinal disorders1/120/140/140/13
Most frequent other events
Showing 10 of 33
Most frequent other events
EventACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth Hormone
PyrexiaGeneral disorders0/120/141/143/13
HeadacheNervous system disorders2/120/141/141/13
AnemiaBlood and lymphatic system disorders0/122/141/140/13
Iron deficiency anemiaBlood and lymphatic system disorders0/120/142/141/13
BronchitisInfections and infestations0/121/142/141/13
HyperthermiaGeneral disorders1/120/140/140/13
Heat exhaustionInjury, poisoning and procedural complications1/120/140/140/13
Procedural dizzinessInjury, poisoning and procedural complications1/120/140/140/13
Ear painEar and labyrinth disorders1/120/140/141/13
Inguinal herniaGastrointestinal disorders1/120/140/140/13

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)ACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth HormoneTotal
<=18 years1214141353
Between 18 and 65 years00000
>=65 years00000
Age, Continuous
Age, Continuous(years)ACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth HormoneTotal
Mean7.98 ± 2.8898.24 ± 2.137.32 ± 2.7847.53 ± 2.4837.77 ± 2.57
Sex: Female, Male
Sex: Female, Male(Participants)ACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth HormoneTotal
Female345315
Male91091038
Race (NIH/OMB)
Race (NIH/OMB)(Participants)ACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth HormoneTotal
American Indian or Alaska Native00000
Asian00000
Native Hawaiian or Other Pacific Islander00000
Black or African American00000
White1214141353
More than one race00000
Unknown or Not Reported00000
Region of Enrollment
Region of Enrollment(participants)ACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth HormoneTotal
Russian Federation447621
Romania10012
Hungary00101
Egypt24017
Ukraine14117
Belarus22217
Greece10203
Turkey10023
Poland00101
Bulgaria00011
Baseline IGF-1 SDS (standard deviation score)
Baseline IGF-1 SDS (standard deviation score)(Standard Deviation Score)ACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth HormoneTotal
Mean-2.034 ± 0.7429-2.017 ± 0.7713-2.19 ± 0.7169-2.502 ± 0.896-2.19 ± 0.782
Baseline Height SDS
Baseline Height SDS(Standard Deviation Score)ACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth HormoneTotal
Mean-3.05 ± 1.127-2.75 ± 0.383-3.17 ± 1.04-3.27 ± 1.077-3.06 ± 0.905
Screening Peak GH Values
Screening Peak GH Values(ng/mL)ACP-001, Dose-level 1ACP-001, Dose-level 2ACP-001, Dose-level 3Human Growth HormoneTotal
Mean5.09 ± 3.1695.16 ± 2.5984.44 ± 2.7705.15 ± 3.0684.95 ± 2.829
07

Study locations

36 sites
  • 2nd Children City Clinic
    Minsk, Belarus
  • University Multiprofile Hospital for Active Treatment (UMHAT) "Sv. Marina"
    Varna, Bulgaria
  • Masaryk´s Hospital
    Ústí nad Labem, Czechia
  • El Shatby University Hospital
    Alexandria, Egypt
  • Ain Shams University Hospital
    Cairo, Egypt
  • Cairo University Hospital
    Cairo, Egypt
  • El Mansoura University Hospital
    El Mansoura, Egypt
  • Hôpital des enfants Pellegrin
    Bordeaux, France
  • Hôpital Jeanne de Flandre
    Lille, France
  • Hôpital Femme-Mère-Enfant
    Lyon, France
  • University Hospital Leipzig
    Leipzig, Germany
  • University Children's Hospital Magdeburg
    Magdeburg, Germany
  • Children's Hospital of Athens "P. A. Kyriakou"
    Athens, Greece
  • Buda Children's Hospital
    Budapest, Hungary
  • Heim Pal Children's Hospital
    Budapest, Hungary
  • University of Pecs
    Pécs, Hungary
  • University of Szeged
    Szeged, Hungary
  • University Medical Hospital
    Katowice, Poland
  • Medical University of Lublin
    Lublin, Poland
  • Regional Hospital N°2 Rzeszow
    Rzeszów, Poland
  • Children's Memorial Health Institute Warsaw
    Warsaw, Poland
  • Emergency Clinical Hospital Cluj-Napoca
    Cluj-Napoca, Romania
  • St. Spiridon County Clinic Emergency Hospital
    Iași, Romania
  • Louis Turcanu Emergency Hospital for Children Timisoara
    Timișoara, Romania
  • Federal State Budgetary Institution
    Moscow, Russia
  • St. Petersburg State Pediatric Medical Academy
    Saint Petersburg, Russia
  • Samara State Medical University
    Samara, Russia
  • Bashkir State Medical University
    Ufa, Russia
  • Children's University Hospital
    Ljubljana, Slovenia
  • Ankara University School of Medicine
    Ankara, Turkey (Türkiye)
  • Ege Üniversity
    Izmir, Turkey (Türkiye)
  • Donetsk Regional Children Clinical Hospital
    Donetsk, Ukraine
  • Kharkiv National Medical University
    Kharkiv, Ukraine
  • Institute of Endocrinology and Metabolism
    Kiev, Ukraine
  • Ukrainian Children Specialized Clinical Hospital
    Kiev, Ukraine
  • Odessa National Medical University
    Odesa, Ukraine
08

References and documents

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 ↗
09

Registry details

Key details

Study ID
NCT01947907
Lead sponsor
Ascendis Pharma A/S
Responsible party
Sponsor
First posted
Sep 23, 2013
Start date
Jul 2013
Primary completion
Jul 2015
Completion
Sep 2015
Results posted
Jan 19, 2017
Last update
Jul 8, 2026

Study contacts

Medical Director, MD
study director · Ascendis Pharma A/S

Oversight

Data monitoring committee
Yes
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