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Completed

NCT Number: NCT01947907

Safety, PK/PD (Pharmacokinetics/Pharmacodynamics) and Efficacy of ACP-001 Weekly Versus Daily hGH in 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.

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Key information

Conditions

Growth Hormone Deficiency (GHD) Bone Diseases Bone Diseases, Developmental Bone Diseases, Endocrine Brain Diseases Central Nervous System Diseases Dwarfism Dwarfism, Pituitary Endocrine System Diseases Hypopituitarism Hypothalamic Diseases Musculoskeletal Diseases Nervous System Diseases Pituitary Diseases

Age range

3 year–12 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

2nd Children City Clinic, Minsk, Belarus

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

  • 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

Treatment and study plan

ACP-001

Drug

Once weekly subcutaneous injection

Human Growth Hormone

Drug

Once daily subcutaneous injection of human Growth Hormone

Other names: Somatropin

Primary outcomes

  1. Incidence of Anti-hGH Binding Antibody Formation

    Time frame: Visit 2 - Visit 5

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

  2. Incidence of Anti-hGH Neutralizing Antibody Formation

    Time frame: Visit 2 - Visit 5

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

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

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

    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.

  4. Cmax of hGH

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

    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)

  5. AUC0-168h of hGH

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

    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)

  6. E-Trough of IGF-1

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

    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

  7. Emax of IGF-1

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

    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

  8. AUEC0-168h of IGF-1

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

    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

Secondary outcomes

  1. Annualized Height Velocity

    Time frame: Baseline to 6 months (Visit 5)

    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

Sponsors and collaborators

Lead sponsor

Ascendis Pharma A/S

Industry

Registry information

Official study 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)

Important dates

Study start
2013
Primary completion
2015
Study completion
2015
First posted
Sep 23, 2013
Registry last updated
Jul 8, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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