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A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)

This study is currently recruiting participants.
Verified November 2016 by Ascendis Pharma A/S
Sponsor:
ClinicalTrials.gov Identifier:
NCT02781727
First Posted: May 24, 2016
Last Update Posted: December 2, 2016
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Ascendis Pharma A/S
  Purpose
A 52 week trial of TransCon hGH, a long-acting growth hormone product, versus human growth hormone therapy. TransCon hGH will be given once-a-week, human growth hormone (hGH) will be given daily. Approximately 150 prepubertal, hGH-treatment naïve children (males and females) with GHD will be included. Randomization will occur in a 2:1 ratio (TransCon hGH : Genotropin). This is a global trial that will be conducted in, but not limited to, the United States, Canada, Germany, France, Poland, Bulgaria, Russia and Australia.

Condition Intervention Phase
Growth Hormone Deficiency, Pediatric hGH (Human Growth Hormone) Endocrine System Diseases Hormones Pituitary Diseases Drug: Once weekly subcutaneous injection of TransCon hGH Drug: Once daily subcutaneous injection of Genotropin Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Phase 3, Randomized, Open-label, Active-controlled, Parallel-group Trial Investigating the Safety, Tolerability, and Efficacy of TransCon hGH Administered Once a Week Versus Standard Daily hGH Replacement Therapy Over 52 Weeks in Prepubertal Children With Growth Hormone Deficiency (GHD)

Resource links provided by NLM:


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

Primary Outcome Measures:
  • Annualized height velocity at 52 weeks for weekly TransCon hGH treatment and the daily hGH treatment groups [ Time Frame: 52 weeks ]
  • Incidence of adverse events [ Time Frame: 52 weeks ]

Secondary Outcome Measures:
  • Annualized height velocity for the TransCon hGH and the daily hGH treatment groups over 52 weeks [ Time Frame: 52 Weeks ]
  • Change in height SDS over 52 weeks for the TransCon hGH and the daily hGH treatment group [ Time Frame: 52 Weeks ]
  • Local Tolerability [ Time Frame: 52 Weeks ]
  • Incidence of anti-hGH antibodies including neutralizing antibodies [ Time Frame: 52 Weeks ]

Estimated Enrollment: 150
Study Start Date: August 2016
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: November 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: TransCon hGH
Once weekly subcutaneous injection of TransCon hGH
Drug: Once weekly subcutaneous injection of TransCon hGH
Once weekly subcutaneous injection
Active Comparator: human growth hormone (Genotropin)
Once daily subcutaneous injection of Genotropin
Drug: Once daily subcutaneous injection of Genotropin
Once daily subcutaneous injection

  Eligibility

Information from the National Library of Medicine

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

Inclusion Criteria:

  • Prepubertal children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage 1 (Tanner 1982) aged:

    • Boys: 3-12 years, inclusive
    • Girls: 3-11 years, inclusive
  • Impaired height (HT) defined as at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS ≤ -2.0) according to the 2000 CDC Growth Charts for the United States Methods and Development, available at http://www.cdc.gov/growthcharts/
  • Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH level of ≤10 ng/mL, determined with a validated assay
  • Bone age (BA) at least 6 months less than chronological age
  • Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1)
  • Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject (if the subject is able to read, understand, and sign)

Exclusion Criteria:

  • Children with a body weight below 12 kg
  • Prior exposure to recombinant hGH or IGF-1 therapy
  • Children with past or present intracranial tumor growth as confirmed by a sellar MRI scan (with contrast) at Screening (MRI results from up to 6 months prior to Screening may be accepted)
  • Children with psychosocial dwarfism
  • Children with idiopathic short stature
  • History or presence of malignant disease; any evidence of present tumor growth
  • Closed epiphyses
  • Major medical conditions and/or presence of contraindication to hGH treatment
  • Participation in any other trial of an investigational agent within 3 months prior to Screening
  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): NCT02781727


Contacts
Contact: Michael Beckert, MD (+49)172-155-2596 mb@ascendispharma.com
Contact: David B Karpf, MD 1-650-352-8361 dbk@ascendispharma.com

Locations
United States, Colorado
Rocky Mountain Pediatric Endocrinology Recruiting
Centennial, Colorado, United States, 80112
Sponsors and Collaborators
Ascendis Pharma A/S
Investigators
Study Director: Michael Beckert, MD Ascendis Pharma A/S
Study Director: David B Karpf, MD Ascendis Pharma, Inc.
  More Information

Responsible Party: Ascendis Pharma A/S
ClinicalTrials.gov Identifier: NCT02781727     History of Changes
Other Study ID Numbers: TransCon hGH CT-301
2016-001145-11 ( EudraCT Number )
First Submitted: May 19, 2016
First Posted: May 24, 2016
Last Update Posted: December 2, 2016
Last Verified: November 2016

Keywords provided by Ascendis Pharma A/S:
Human Growth Hormone
hGH
GHD
rHGH
Pediatric Growth Hormone Deficiency
Long Acting Growth Hormone
Somatropin
Prodrug
Growth Failure
Growth Hormone Replacement Therapy
Sustained Release
Sustained Release Growth Hormone
Growth Hormone Deficiency

Additional relevant MeSH terms:
Endocrine System Diseases
Dwarfism, Pituitary
Pituitary 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