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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)

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.
 
ClinicalTrials.gov Identifier: NCT02781727
Recruitment Status : Completed
First Posted : May 24, 2016
Last Update Posted : December 19, 2019
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S

Tracking Information
First Submitted Date  ICMJE May 19, 2016
First Posted Date  ICMJE May 24, 2016
Last Update Posted Date December 19, 2019
Actual Study Start Date  ICMJE December 13, 2016
Actual Primary Completion Date January 17, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 23, 2018)
Annualized height velocity at 52 weeks for weekly TransCon hGH treatment and the daily hGH treatment groups [ Time Frame: 52 weeks ]
Original Primary Outcome Measures  ICMJE
 (submitted: May 20, 2016)
  • 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 ]
  • Local tolerability [ Time Frame: 52 weeks ]
  • Incidence of anti-hGH antibodies including neutralizing antibodies [ Time Frame: 52 weeks ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 23, 2018)
  • Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 52 Weeks ]
  • Annualized height velocity for the TransCon hGH and the daily hGH treatment groups over 52 weeks [ Time Frame: 52 Weeks ]
  • Change in height standard deviation score (SDS) over 52 weeks for the TransCon hGH and the daily hGH treatment groups [ Time Frame: 52 Weeks ]
  • Evaluate serum IGF-1 and IGFBP-3 and IGF-1 SDS and IGFBP-3 SDS over 52 weeks for the TransCon hGH and the daily hGH treatment groups [ Time Frame: 52 Weeks ]
  • Incidence of anti-hGH antibodies including neutralizing antibodies [ Time Frame: 52 Weeks ]
Original Secondary Outcome Measures  ICMJE
 (submitted: May 20, 2016)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)
Official Title  ICMJE 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)
Brief Summary 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Growth Hormone Deficiency, Pediatric
  • hGH (Human Growth Hormone)
  • Endocrine System Diseases
  • Hormones
  • Pituitary Diseases
Intervention  ICMJE
  • Drug: Once weekly subcutaneous injection of TransCon hGH
    Once weekly subcutaneous injection
  • Drug: Once daily subcutaneous injection of Genotropin
    Once daily subcutaneous injection
Study Arms  ICMJE
  • Experimental: TransCon hGH
    Once weekly subcutaneous injection of TransCon hGH
    Intervention: Drug: Once weekly subcutaneous injection of TransCon hGH
  • Active Comparator: human growth hormone (Genotropin)
    Once daily subcutaneous injection of Genotropin
    Intervention: Drug: Once daily subcutaneous injection of Genotropin
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 3, 2018)
161
Original Estimated Enrollment  ICMJE
 (submitted: May 20, 2016)
150
Actual Study Completion Date  ICMJE January 17, 2019
Actual Primary Completion Date January 17, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Armenia,   Australia,   Belarus,   Bulgaria,   Georgia,   Greece,   Italy,   New Zealand,   Poland,   Romania,   Russian Federation,   Turkey,   Ukraine,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02781727
Other Study ID Numbers  ICMJE TransCon hGH CT-301
2016-001145-11 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Ascendis Pharma A/S
Study Sponsor  ICMJE Ascendis Pharma A/S
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Michael Beckert, MD Ascendis Pharma A/S
Study Director: Aimee D Shu, MD Ascendis Pharma, Inc.
PRS Account Ascendis Pharma A/S
Verification Date December 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP