We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    NCT02781727
Previous Study | Return to List | Next Study

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
Results First Posted : January 4, 2022
Last Update Posted : January 4, 2022
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S ( Ascendis Pharma Endocrinology Division A/S )

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 Armenia, Australia, Belarus, Bulgaria, Georgia, Greece, Italy, New Zealand, Poland, Romania, Russia, Turkey, Ukraine, and the United States.

Condition or disease Intervention/treatment 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

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 162 participants
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)
Actual Study Start Date : December 13, 2016
Actual Primary Completion Date : January 17, 2019
Actual Study Completion Date : January 17, 2019


Arm Intervention/treatment
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




Primary Outcome Measures :
  1. Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups [ Time Frame: 52 weeks ]
    Annualized height velocity (AHV) at 52 weeks for weekly lonapegsomatropin (TransCon hGH) and daily hGH treatment groups


Secondary Outcome Measures :
  1. Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 52 Weeks ]
    Number of participants with Treatment-Emergent Adverse Events for the weekly lonapegsomatropin and daily hGH treatment groups

  2. Annualized Height Velocity Over 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups [ Time Frame: Week 5, Week 13, Week 26, Week 39 and Week 52 ]
    Annualized height velocity (AHV) over 52 weeks for weekly lonapegsomatropin and daily hGH treatment groups. AHV by visit was determined by ANCOVA model with multiple imputation. For each imputed data set, an ANCOVA model with by visit AHV as the dependent variable, treatment and gender as factors, baseline age, baseline peak GH levels (log transformed) at stimulation test, and baseline height SDS - average parental height SDS as covariates were fitted.

  3. Change in Height Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups [ Time Frame: Week 5, Week 13, Week 26, Week 39 and Week 52 ]
    Height Standard Deviation Score (SDS) is the number of standard deviations above or below the mean height for age and sex. Height SDS was derived using the LMS method as ((Height/M)^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from 2000 CDC growth charts for the United States. A Standard Deviation Score of 0 represents the population mean. A higher change from baseline in Height Standard Deviation Score (SDS) indicates a better outcome. The change from baseline in height SDS by visit was determined by ANCOVA model and included baseline age, peak GH levels (log transformed) at stimulation test and baseline height SDS as covariates, as well as treatment and gender as factors.

  4. Average IGF-1 Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups [ Time Frame: Week 13, Week 26, Week 39, and Week 52 ]
    IGF-1 Standard Deviation Score (SDS) is the number of standard deviations above or below the mean Insulin-like Growth Factor 1 (IGF-1) level for age and sex. IGF-1 SDS was derived using the LMS method as ((IGF-1/M)^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from Bidlingmaier et al. (2014). A Standard Deviation Score of 0 represents the population mean. Average IGF-1 SDS by visit was determined by ANCOVA. The ANCOVA model included baseline age, peak GH levels (log transformed) at stimulation test, baseline IGF-1 SDS as covariates, as well as treatment and gender as factors. Modeled values begin at Week 13 corresponding with achievement of IGF-1 steady state. Average IGF-1 SDS values by visit for the Lonapegsomatropin group were derived from a population pharmacodynamic model; the average IGF-1 SDS values for the Genotropin group are represented by observed values.

  5. Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation [ Time Frame: Start of study treatment through Week 52 ]
    Number of participants with treatment emergent anti-hGH binding antibody formation during the 52 week study. All samples were negative for anti-hGH neutralizing antibodies.



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.


Layout table for eligibility information
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

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


Locations
Show Show 53 study locations
Sponsors and Collaborators
Ascendis Pharma Endocrinology Division A/S
Investigators
Layout table for investigator information
Study Director: Michael Beckert, MD Ascendis Pharma A/S
Study Director: Aimee D Shu, MD Ascendis Pharma, Inc.
  Study Documents (Full-Text)

Documents provided by Ascendis Pharma A/S ( Ascendis Pharma Endocrinology Division A/S ):
Study Protocol  [PDF] September 12, 2017
Statistical Analysis Plan  [PDF] February 19, 2019

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Ascendis Pharma Endocrinology Division A/S
ClinicalTrials.gov Identifier: NCT02781727    
Other Study ID Numbers: TransCon hGH CT-301
2016-001145-11 ( EudraCT Number )
First Posted: May 24, 2016    Key Record Dates
Results First Posted: January 4, 2022
Last Update Posted: January 4, 2022
Last Verified: December 2021
Keywords provided by Ascendis Pharma A/S ( Ascendis Pharma Endocrinology Division 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:
Layout table for MeSH terms
Dwarfism, Pituitary
Pituitary Diseases
Endocrine System Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism