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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03344458
Registration number
NCT03344458
Ethics application status
Date submitted
14/11/2017
Date registered
17/11/2017
Date last updated
8/05/2024
Titles & IDs
Public title
A Long-Term Trial Investigating Safety and Efficacy of TransCon hGH in Children With Growth Hormone Deficiency Who Have Completed a Prior TransCon hGH Clinical Trial
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Scientific title
enliGHten: A Multicenter, Phase 3, Long-Term, Open-Label Trial Investigating Safety and Efficacy of TransCon hGH Administered Once-Weekly in Children With Growth Hormone Deficiency (GHD) Who Have Completed a Prior TransCon hGH Clinical Trial
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Secondary ID [1]
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2017-003410-20
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Secondary ID [2]
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TransCon hGH CT-301EXT
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Universal Trial Number (UTN)
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Trial acronym
enliGHten
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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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Endocrine System Diseases
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Hormone Deficiency
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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 - TransCon hGH
Experimental: TransCon hGH - Once weekly subcutaneous injection of TransCon hGH
Treatment: Drugs: TransCon hGH
Once weekly subcutaneous injection of TransCon hGH
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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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Number of Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability]
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Assessment method [1]
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Long-term safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment
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Timepoint [1]
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Up to Week 208
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Secondary outcome [1]
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Annualized Height Velocity by Visit
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Assessment method [1]
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Annualized height velocity (AHV) by visit with long-term dosing of weekly lonapegsomatropin treatment
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Timepoint [1]
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Up to Week 208
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Secondary outcome [2]
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IGF-1 SDS by Visit
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Assessment method [2]
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Insulin-like Growth Factor-1 (IGF-1) standard deviation score (SDS) by visit with long-term dosing of weekly lonapegsomatropin treatment at Week 13, Week 52, Week 104, Week 156, and Week 208. IGF-1 SDS is the number of standard deviations above or below the mean IGF-1 level for a child of the same age and sex. The target range for IGF-1 SDS was 0 to +2. An IGF-1 SDS of 0 represents the population mean. If the IGF-1 SDS is < 0, then the negative score indicates an IGF-1 below the mean IGF-1 for a child of the same age and sex. If the IGF-1 SDS is > 0, then the positive score indicates an IGF-1 above the mean IGF-1 for a child of the same age and sex. A positive score of IGF-1 SDS indicates a better outcome.
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Timepoint [2]
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Up to Week 208
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Secondary outcome [3]
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Height SDS - Change From Baseline
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Assessment method [3]
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Change in Height Standard Deviation Score (SDS) from baseline with long-term dosing of weekly lonapegsomatropin treatment at Week 13, Week 52, Week 104, Week 156, and Week 208. Height SDS is the number of standard deviations above or below the mean height for age and sex. A Standard Deviation Score of 0 equals the population mean. If the Height SDS is < 0, then the negative score indicates a height below the mean height for a child of the same age and sex. If the Height SDS is > 0, then the positive score indicates a height above the mean height for a child of the same age and sex. A positive change from baseline in Height SDS indicates improved outcomes.
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Timepoint [3]
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Up to Week 208
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Eligibility
Key inclusion criteria
1. Children who have completed a prior phase 3 TransCon hGH trial
2. Children who have not permanently discontinued study drug in the prior trial
3. Written, signed, informed consent of the parent or legal guardian of the subject and
written assent of the subject as required by the IRB/HREC/IEC
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Minimum age
1
Year
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Maximum age
18
Years
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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
1. Poorly-controlled diabetes mellitus (HbA1c = 8.0%) or diabetic complications
2. Evidence of closed epiphyses, defined as bone age > 14.0 years for females or > 16.0
years for males
3. Major medical conditions unless approved by Medical Expert
4. Known hypersensitivity to the components of the trial medication
5. Likely to be non-compliant with respect to trial conduct (in regards to the subject
and/or the parent/legal guardian/caregiver)
6. Pregnancy
7. Any other reason that in the opinion of the investigator would prevent the subject
from completing participation or following the trial schedule
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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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
Single group
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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
19/12/2017
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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
21/02/2023
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Sample size
Target
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Accrual to date
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Final
298
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Recruitment in Australia
Recruitment state(s)
VIC
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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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Country [2]
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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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Country [5]
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United States of America
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State/province [5]
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Minnesota
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Country [6]
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United States of America
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State/province [6]
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Mississippi
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Country [7]
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United States of America
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State/province [7]
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New Hampshire
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Country [8]
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United States of America
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State/province [8]
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New York
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United States of America
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State/province [9]
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Ohio
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United States of America
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State/province [10]
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Oklahoma
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United States of America
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State/province [11]
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Oregon
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Country [12]
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United States of America
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State/province [12]
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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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Virginia
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Country [14]
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United States of America
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State/province [14]
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Washington
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Country [15]
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Armenia
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State/province [15]
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Yerevan
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Country [16]
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Belarus
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State/province [16]
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Minsk
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Country [17]
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Bulgaria
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State/province [17]
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Varna
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Country [18]
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Georgia
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State/province [18]
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Tbilisi
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Greece
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State/province [19]
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Athens
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New Zealand
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Auckland
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Poland
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State/province [21]
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Gdansk
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Poland
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State/province [22]
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Warsaw
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Country [23]
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Russian Federation
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State/province [23]
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Izhevsk
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Country [24]
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Russian Federation
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State/province [24]
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Krasnoyarsk
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Russian Federation
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State/province [25]
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Moscow
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Russian Federation
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State/province [26]
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Saint Petersburg
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Russian Federation
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State/province [27]
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Saratov
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Russian Federation
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State/province [28]
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Ufa
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Country [29]
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Russian Federation
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State/province [29]
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Vologda
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Russian Federation
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State/province [30]
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Voronezh
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Country [31]
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Ukraine
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State/province [31]
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Kharkiv
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Country [32]
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Ukraine
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State/province [32]
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Kyiv
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Country [33]
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Ukraine
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State/province [33]
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Odesa
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Ascendis Pharma A/S
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
A multicenter, phase 3, long-term extension trial of TransCon hGH administered once-weekly in
children with growth hormone deficiency (GHD) who previously participated in a phase 3
TransCon hGH trial. Approximately 300 children (males and females) with GHD will be included.
All study participants will receive TransCon hGH. This is a global trial that will be
conducted in, but not limited to, the United States, Poland, Bulgaria, Ukraine, Armenia,
Russia and Australia.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03344458
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Trial related presentations / publications
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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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Country
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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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Address
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03344458
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