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

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by Ascendis Pharma A/S
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S
ClinicalTrials.gov Identifier:
NCT01947907
First received: June 3, 2013
Last updated: July 3, 2014
Last verified: July 2014
  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
Endpoint Classification: Pharmacokinetics/Dynamics Study
Intervention Model: Parallel Assignment
Masking: 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:
  • Safety of ACP-001 on different dosing levels [ Time Frame: 26 weeks ] [ Designated as safety issue: Yes ]
    Number of adverse events

  • Pharmacokinetics of ACP-001 [ Time Frame: 26 weeks ] [ Designated as safety issue: No ]
    Pharmacokinetic assessment of ACP-001 by AUC

  • IGF-I response [ Time Frame: 26 weeks ] [ Designated as safety issue: Yes ]
    IGF-I levels and change in IGF-I levels over a period of 26 weeks

  • Local tolerability of ACP-001 [ Time Frame: 26 weeks ] [ Designated as safety issue: Yes ]
    Number of injection site reactions

  • Safety of ACP-001 on different dosing levels [ Time Frame: 26 weeks ] [ Designated as safety issue: Yes ]
    Incidence of anti-hGH antibodies


Secondary Outcome Measures:
  • Height velocity [ Time Frame: 26 weeks ] [ Designated as safety issue: No ]
    Height velocity over a period of 26 weeks (annualized)


Estimated Enrollment: 52
Study Start Date: April 2013
Estimated Primary Completion Date: May 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

Ages Eligible for Study:   3 Years to 12 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Prepubertal children, Tanner stage 1
  • Diagnosis of GHD, confirmed by two stimulation tests
  • Bon age not greater than chronological age
  • Impaired height and height velocity
  • BMI within +/- 2 SD
  • Baseline IGF-I
  • 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
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01947907

Contacts
Contact: Michael Beckert, MD +4536944486 mb@ascendispharma.com

  Show 32 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

No publications provided

Responsible Party: Ascendis Pharma A/S
ClinicalTrials.gov Identifier: NCT01947907     History of Changes
Other Study ID Numbers: ACP-001_CT-004
Study First Received: June 3, 2013
Last Updated: July 3, 2014
Health Authority: Germany: Federal Institute for Drugs and Medical Devices
Hungary: Ministry of Health, Social and Family Affairs
Czech Republic: State Institute for Drug Control

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

Additional relevant MeSH terms:
Pituitary Diseases
Dwarfism, Pituitary
Endocrine System Diseases
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 22, 2014