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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02781727
Registration number
NCT02781727
Ethics application status
Date submitted
19/05/2016
Date registered
24/05/2016
Date last updated
4/01/2022
Titles & IDs
Public title
A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)
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Scientific 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)
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Secondary ID [1]
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2016-001145-11
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Secondary ID [2]
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TransCon hGH CT-301
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Growth Hormone Deficiency, Pediatric
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hGH (Human Growth Hormone)
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Endocrine System Diseases
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Hormones
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Pituitary Diseases
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Condition category
Condition code
Metabolic and Endocrine
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Other endocrine disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Other neurological disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Once weekly subcutaneous injection of TransCon hGH
Treatment: Drugs - Once daily subcutaneous injection of Genotropin
Experimental: TransCon hGH - Once weekly subcutaneous injection of TransCon hGH
Active comparator: human growth hormone (Genotropin) - Once daily subcutaneous injection of Genotropin
Treatment: Drugs: Once weekly subcutaneous injection of TransCon hGH
Once weekly subcutaneous injection
Treatment: Drugs: Once daily subcutaneous injection of Genotropin
Once daily subcutaneous injection
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
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Assessment method [1]
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Annualized height velocity (AHV) at 52 weeks for weekly lonapegsomatropin (TransCon hGH) and daily hGH treatment groups
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Timepoint [1]
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52 weeks
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Secondary outcome [1]
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Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
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Assessment method [1]
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Number of participants with Treatment-Emergent Adverse Events for the weekly lonapegsomatropin and daily hGH treatment groups
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Timepoint [1]
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52 Weeks
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Secondary outcome [2]
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Annualized Height Velocity Over 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
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Assessment method [2]
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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.
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Timepoint [2]
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Week 5, Week 13, Week 26, Week 39 and Week 52
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Secondary outcome [3]
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Change in Height Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups
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Assessment method [3]
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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.
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Timepoint [3]
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Week 5, Week 13, Week 26, Week 39 and Week 52
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Secondary outcome [4]
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Average IGF-1 Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups
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Assessment method [4]
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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.
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Timepoint [4]
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Week 13, Week 26, Week 39, and Week 52
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Secondary outcome [5]
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Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation
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Assessment method [5]
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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.
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Timepoint [5]
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Start of study treatment through Week 52
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Eligibility
Key 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)
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Minimum age
3
Years
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Maximum age
12
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
13/12/2016
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
17/01/2019
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Sample size
Target
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Accrual to date
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Final
162
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Ascendis Pharma Investigational Site - Clayton
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Recruitment postcode(s) [1]
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3168 - Clayton
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Alabama
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United States of America
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Arkansas
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United States of America
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California
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United States of America
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Colorado
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United States of America
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Florida
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United States of America
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Minnesota
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United States of America
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State/province [7]
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Mississippi
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United States of America
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New Hampshire
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United States of America
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New York
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United States of America
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Oklahoma
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United States of America
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Oregon
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United States of America
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Texas
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Country [13]
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United States of America
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State/province [13]
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Washington
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Country [14]
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Armenia
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State/province [14]
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Yerevan
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Country [15]
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Belarus
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State/province [15]
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Minsk
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Bulgaria
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State/province [16]
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Varna
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Georgia
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Tbilisi
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Greece
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Athens
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Italy
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Milano
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Italy
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Roma
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New Zealand
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Grafton
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Poland
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Gdansk
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Poland
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Warszawa
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Romania
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Iasi
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Russian Federation
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Izhevsk
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Russian Federation
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Kazan
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Russian Federation
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Krasnoyarsk
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Russian Federation
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Moscow
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Russian Federation
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Nizhny Novgorod
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Russian Federation
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Novosibirsk
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Russian Federation
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Omsk
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Russian Federation
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Saint Petersburg
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Russian Federation
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Samara
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Russian Federation
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Saratov
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Russian Federation
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Tomsk
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Russian Federation
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Ufa
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Russian Federation
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Vologda
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Russian Federation
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Voronezh
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Turkey
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Izmir
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Turkey
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Melikgazi
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Turkey
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Trabzon
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Ukraine
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Kharkov
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Ukraine
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Kyiv
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Ukraine
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State/province [44]
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Odesa
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Ascendis Pharma Endocrinology Division A/S
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Address
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/study/NCT02781727
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Trial related presentations / publications
Thornton PS, Maniatis AK, Aghajanova E, Chertok E, Vlachopapadopoulou E, Lin Z, Song W, Christoffersen ED, Breinholt VM, Kovalenko T, Giorgadze E, Korpal-Szczyrska M, Hofman PL, Karpf DB, Shu AD, Beckert M. Weekly Lonapegsomatropin in Treatment-Naive Children With Growth Hormone Deficiency: The Phase 3 heiGHt Trial. J Clin Endocrinol Metab. 2021 Oct 21;106(11):3184-3195. doi: 10.1210/clinem/dgab529.
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Public notes
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Contacts
Principal investigator
Name
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Michael Beckert, MD
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Address
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Ascendis Pharma A/S
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Fax
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Email
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/27/NCT02781727/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/27/NCT02781727/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02781727
Download to PDF