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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02781727




Registration number
NCT02781727
Ethics application status
Date submitted
19/05/2016
Date registered
24/05/2016
Date last updated
19/04/2019

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 TransCon hGH treatment and the daily hGH treatment groups
Timepoint [1] 0 0
52 weeks
Secondary outcome [1] 0 0
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Timepoint [1] 0 0
52 Weeks
Secondary outcome [2] 0 0
Annualized height velocity for the TransCon hGH and the daily hGH treatment groups over 52 weeks
Timepoint [2] 0 0
52 Weeks
Secondary outcome [3] 0 0
Change in height standard deviation score (SDS) over 52 weeks for the TransCon hGH and the daily hGH treatment groups
Timepoint [3] 0 0
52 Weeks
Secondary outcome [4] 0 0
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
Timepoint [4] 0 0
52 Weeks
Secondary outcome [5] 0 0
Incidence of anti-hGH antibodies including neutralizing antibodies
Timepoint [5] 0 0
52 Weeks

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
Gender
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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 A/S
Address
Country

Ethics approval
Ethics application status

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, but not limited to, the United
States, Canada, Germany, France, Poland, Bulgaria, Russia and Australia.
Trial website
https://clinicaltrials.gov/show/NCT02781727
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

Summary results
Other publications