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/NCT03305016




Registration number
NCT03305016
Ethics application status
Date submitted
4/10/2017
Date registered
9/10/2017
Date last updated
23/05/2019

Titles & IDs
Public title
A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone Deficiency
Scientific title
fliGHt: A Multicenter, Phase 3, Open-Label, 26-Week Trial Investigating the Safety, Tolerability and Efficacy of TransCon hGH Administered Once Weekly in Children With GHD
Secondary ID [1] 0 0
U1111-1199-8218
Secondary ID [2] 0 0
TransCon hGH CT-302
Universal Trial Number (UTN)
Trial acronym
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 at a starting dose of 0.24 mg/kg/week

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

Outcomes
Primary outcome [1] 0 0
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] - Safety and tolerability of weekly TransCon hGH treatment
Timepoint [1] 0 0
26 weeks
Secondary outcome [1] 0 0
Annualized height velocity at 26 weeks of weekly TransCon hGH treatment
Timepoint [1] 0 0
26 weeks
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 at 26 weeks of weekly TransCon hGH treatment
Timepoint [2] 0 0
26 weeks
Secondary outcome [3] 0 0
Change in height standard deviation scores at 26 weeks of weekly TransCon hGH treatment
Timepoint [3] 0 0
26 weeks
Secondary outcome [4] 0 0
Incidence of antibodies against TransCon hGH over 26 weeks of weekly TransCon hGH treatment
Timepoint [4] 0 0
26 weeks

Eligibility
Key inclusion criteria
1. Investigator-determined GHD diagnosis prior to the historical initiation of daily hGH
therapy.

2. 6 months to 17 years old, inclusive, at Visit 1

1. If 3 to 17 years old, are taking daily hGH at a dose of = 0.20 mg hGH/kg/week for
at least 13 weeks but no more than 130 weeks prior to Visit 1

2. If = 6 months but < 3 years old, are either hGH treatment-naïve or are taking
daily hGH at a dose of = 0.20mg hGH/kg/week for no more than 130 weeks prior to
Visit 1

3. Tanner stage < 5 at Visit 1

4. Open epiphyses (bone age =14.0 years for females or =16.0 years for males)

5. 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
6 Months
Maximum age
17 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Weight of < 5.5 kg or > 80 kg at Visit 1

2. Females of child-bearing potential

3. History of malignant disease

4. Any clinically significant abnormality likely to affect growth or the ability to
evaluate growth (eg, chronic diseases or conditions such as renal insufficiency,
spinal cord irradiation, hypothyroidism, active celiac disease, malnutrition or
psychosocial dwarfism)

5. Poorly-controlled diabetes mellitus (HbA1c >8.0%) or diabetic complications

6. Known neutralizing antibodies against hGH

7. Major medical conditions, unless approved by Medical Monitor

8. Pregnancy

9. Presence of contraindications to hGH treatment

10. Likely to be non-compliant with respect to trial conduct (in regards to the subject
and/or the parent/legal guardian/caregiver)

11. Participation in any other trial of an investigational agent within 30 days prior to
Visit 1

12. Prior exposure to investigational hGH

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
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)
VIC
Recruitment hospital [1] 0 0
Monash Children's Hospital - 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
Iowa
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
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Oklahoma
Country [12] 0 0
United States of America
State/province [12] 0 0
Oregon
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
United States of America
State/province [14] 0 0
Virginia
Country [15] 0 0
Canada
State/province [15] 0 0
Alberta
Country [16] 0 0
New Zealand
State/province [16] 0 0
Auckland

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

Ethics approval
Ethics application status

Summary
Brief summary
A 26 week trial of TransCon hGH, a long-acting growth hormone product, administered
once-a-week. Approximately 150 children (males and females) with growth hormone deficiency
(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, Canada, Australia,
and New Zealand.
Trial website
https://clinicaltrials.gov/show/NCT03305016
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Aimee D Shu, MD
Address 0 0
Ascendis Pharma, Inc.
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