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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03344458




Registration number
NCT03344458
Ethics application status
Date submitted
14/11/2017
Date registered
17/11/2017
Date last updated
10/01/2019

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
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
Secondary ID [1] 0 0
2017-003410-20
Secondary ID [2] 0 0
TransCon hGH CT-301EXT
Universal Trial Number (UTN)
Trial acronym
enliGHten
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Growth Hormone Deficiency, Pediatric 0 0
Endocrine System Diseases 0 0
Hormone Deficiency 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 - TransCon hGH

Experimental: TransCon hGH - Once weekly subcutaneous injection of TransCon hGH


Treatment: Drugs: TransCon hGH
Once weekly subcutaneous injection of TransCon hGH

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability] - Long-term safety and tolerability of weekly TransCon hGH treatment
Timepoint [1] 0 0
Up to 4 years
Secondary outcome [1] 0 0
Annualized height velocity (HV) with long-term dosing of weekly TransCon hGH treatment
Timepoint [1] 0 0
Up to 4 years
Secondary outcome [2] 0 0
Proportion of subjects with IGF-1 standard deviation score (SDS) in the normal range of 0.0 to +2.0 with long-term dosing of weekly TransCon hGH treatment
Timepoint [2] 0 0
Up to 4 years
Secondary outcome [3] 0 0
Change in height standard deviation scores with long-term dosing of weekly TransCon hGH treatment
Timepoint [3] 0 0
Up to 4 years
Secondary outcome [4] 0 0
Incidence of antibodies against TransCon hGH with long-term dosing of weekly TransCon hGH treatment
Timepoint [4] 0 0
Up to 4 years

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
Minimum age
1 Year
Maximum age
18 Years
Gender
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
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
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
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)
VIC
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
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
Mississippi
Country [7] 0 0
United States of America
State/province [7] 0 0
New Hampshire
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
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
Virginia
Country [14] 0 0
United States of America
State/province [14] 0 0
Washington
Country [15] 0 0
Armenia
State/province [15] 0 0
Yerevan
Country [16] 0 0
Belarus
State/province [16] 0 0
Minsk
Country [17] 0 0
Bulgaria
State/province [17] 0 0
Varna
Country [18] 0 0
Georgia
State/province [18] 0 0
Tbilisi
Country [19] 0 0
Greece
State/province [19] 0 0
Athens
Country [20] 0 0
New Zealand
State/province [20] 0 0
Auckland
Country [21] 0 0
Poland
State/province [21] 0 0
Gdansk
Country [22] 0 0
Poland
State/province [22] 0 0
Warsaw
Country [23] 0 0
Russian Federation
State/province [23] 0 0
Izhevsk
Country [24] 0 0
Russian Federation
State/province [24] 0 0
Krasnoyarsk
Country [25] 0 0
Russian Federation
State/province [25] 0 0
Moscow
Country [26] 0 0
Russian Federation
State/province [26] 0 0
Saint Petersburg
Country [27] 0 0
Russian Federation
State/province [27] 0 0
Saratov
Country [28] 0 0
Russian Federation
State/province [28] 0 0
Ufa
Country [29] 0 0
Russian Federation
State/province [29] 0 0
Vologda
Country [30] 0 0
Russian Federation
State/province [30] 0 0
Voronezh
Country [31] 0 0
Ukraine
State/province [31] 0 0
Kharkiv
Country [32] 0 0
Ukraine
State/province [32] 0 0
Kyiv
Country [33] 0 0
Ukraine
State/province [33] 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 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.
Trial website
https://clinicaltrials.gov/show/NCT03344458
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
Michael Beckert, MD
Address 0 0
Country 0 0
Phone 0 0
+49 172-155-2596
Fax 0 0
Email 0 0
mb@ascendispharma.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03344458