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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)
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)
Secondary ID [1] 0 0
2016-001145-11
Secondary ID [2] 0 0
TransCon hGH CT-301
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Growth Hormone Deficiency, Pediatric 0 0
hGH (Human Growth Hormone) 0 0
Endocrine System Diseases 0 0
Hormones 0 0
Pituitary Diseases 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Timepoint [1] 0 0
52 weeks
Secondary outcome [1] 0 0
Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
Timepoint [1] 0 0
52 Weeks
Secondary outcome [2] 0 0
Annualized Height Velocity Over 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Timepoint [2] 0 0
Week 5, Week 13, Week 26, Week 39 and Week 52
Secondary outcome [3] 0 0
Change in Height Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Timepoint [3] 0 0
Week 5, Week 13, Week 26, Week 39 and Week 52
Secondary outcome [4] 0 0
Average IGF-1 Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Timepoint [4] 0 0
Week 13, Week 26, Week 39, and Week 52
Secondary outcome [5] 0 0
Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation
Timepoint [5] 0 0
Start of study treatment through Week 52

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)
Minimum age
3 Years
Maximum age
12 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/12/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
17/01/2019
Sample size
Target
Accrual to date
Final
162
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Ascendis Pharma Investigational Site - Clayton
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arkansas
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Colorado
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
Mississippi
Country [8] 0 0
United States of America
State/province [8] 0 0
New Hampshire
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
Oklahoma
Country [11] 0 0
United States of America
State/province [11] 0 0
Oregon
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
United States of America
State/province [13] 0 0
Washington
Country [14] 0 0
Armenia
State/province [14] 0 0
Yerevan
Country [15] 0 0
Belarus
State/province [15] 0 0
Minsk
Country [16] 0 0
Bulgaria
State/province [16] 0 0
Varna
Country [17] 0 0
Georgia
State/province [17] 0 0
Tbilisi
Country [18] 0 0
Greece
State/province [18] 0 0
Athens
Country [19] 0 0
Italy
State/province [19] 0 0
Milano
Country [20] 0 0
Italy
State/province [20] 0 0
Roma
Country [21] 0 0
New Zealand
State/province [21] 0 0
Grafton
Country [22] 0 0
Poland
State/province [22] 0 0
Gdansk
Country [23] 0 0
Poland
State/province [23] 0 0
Warszawa
Country [24] 0 0
Romania
State/province [24] 0 0
Iasi
Country [25] 0 0
Russian Federation
State/province [25] 0 0
Izhevsk
Country [26] 0 0
Russian Federation
State/province [26] 0 0
Kazan
Country [27] 0 0
Russian Federation
State/province [27] 0 0
Krasnoyarsk
Country [28] 0 0
Russian Federation
State/province [28] 0 0
Moscow
Country [29] 0 0
Russian Federation
State/province [29] 0 0
Nizhny Novgorod
Country [30] 0 0
Russian Federation
State/province [30] 0 0
Novosibirsk
Country [31] 0 0
Russian Federation
State/province [31] 0 0
Omsk
Country [32] 0 0
Russian Federation
State/province [32] 0 0
Saint Petersburg
Country [33] 0 0
Russian Federation
State/province [33] 0 0
Samara
Country [34] 0 0
Russian Federation
State/province [34] 0 0
Saratov
Country [35] 0 0
Russian Federation
State/province [35] 0 0
Tomsk
Country [36] 0 0
Russian Federation
State/province [36] 0 0
Ufa
Country [37] 0 0
Russian Federation
State/province [37] 0 0
Vologda
Country [38] 0 0
Russian Federation
State/province [38] 0 0
Voronezh
Country [39] 0 0
Turkey
State/province [39] 0 0
Izmir
Country [40] 0 0
Turkey
State/province [40] 0 0
Melikgazi
Country [41] 0 0
Turkey
State/province [41] 0 0
Trabzon
Country [42] 0 0
Ukraine
State/province [42] 0 0
Kharkov
Country [43] 0 0
Ukraine
State/province [43] 0 0
Kyiv
Country [44] 0 0
Ukraine
State/province [44] 0 0
Odesa

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ascendis Pharma Endocrinology Division A/S
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Michael Beckert, MD
Address 0 0
Ascendis Pharma A/S
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02781727