Trial record 15 of 43 for:    Open Studies | "Dwarfism"

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.
Verified September 2013 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: September 18, 2013
Last verified: September 2013
  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 2014 (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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01947907

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

Locations
Germany
University of Leipzig Not yet recruiting
Leipzig, Germany
University of Magdeburg Recruiting
Magdeburg, Germany
Hungary
University of Budapest Recruiting
Budapest, Hungary
University of Pecs Recruiting
Pecs, Hungary
University of Szeged Recruiting
Szeged, Hungary
Russian Federation
University of Samara Recruiting
Samara, Russian Federation
University of Ufa Recruiting
Ufa, Russian Federation
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: September 18, 2013
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:
Dwarfism, Pituitary
Dwarfism
Endocrine System Diseases
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
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 14, 2014