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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01947907
First Posted: September 23, 2013
Last Update Posted: January 19, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Ascendis Pharma A/S
  Purpose
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.

Condition Intervention Phase
Growth Hormone Deficiency (GHD) Drug: ACP-001 Drug: Human Growth Hormone Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)

Resource links provided by NLM:


Further study details as provided by Ascendis Pharma A/S:

Primary Outcome Measures:
  • 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

  • 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

  • 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.

  • 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)


  • 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)


  • 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


  • 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


  • 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 Outcome Measures:
  • 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


Enrollment: 53
Study Start Date: July 2013
Study Completion Date: September 2015
Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ACP-001, dose-level 1
Once weekly subcutaneous injection of ACP-001
Drug: ACP-001
Once weekly subcutaneous injection
Experimental: ACP-001, dose-level 2
Once weekly subcutaneous injection of ACP-001
Drug: ACP-001
Once weekly subcutaneous injection
Experimental: ACP-001, dose-level 3
Once weekly subcutaneous injection of ACP-001
Drug: ACP-001
Once weekly subcutaneous injection
Active Comparator: Human Growth Hormone
Once daily subcutaneous injection of human Growth Hormone (rhGH)
Drug: Human Growth Hormone
Once daily subcutaneous injection of human Growth Hormone
Other Name: Somatropin

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   3 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01947907


  Show 36 Study Locations
Sponsors and Collaborators
Ascendis Pharma A/S
Investigators
Principal Investigator: Pierre Chatelain, Prof, MD University of Lyon
Study Director: Michael Beckert, MD Ascendis Pharma A/S
  More Information

Responsible Party: Ascendis Pharma A/S
ClinicalTrials.gov Identifier: NCT01947907     History of Changes
Other Study ID Numbers: ACP-001_CT-004
First Submitted: June 3, 2013
First Posted: September 23, 2013
Results First Submitted: October 3, 2016
Results First Posted: January 19, 2017
Last Update Posted: January 19, 2017
Last Verified: November 2016

Keywords provided by Ascendis Pharma A/S:
Human Growth Hormone
rhGH
hGH
GHD

Additional relevant MeSH terms:
Endocrine System Diseases
Dwarfism, Pituitary
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
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs