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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03305016
Registration number
NCT03305016
Ethics application status
Date submitted
4/10/2017
Date registered
9/10/2017
Date last updated
4/01/2022
Titles & IDs
Public title
A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone Deficiency
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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
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Secondary ID [1]
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U1111-1199-8218
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Secondary ID [2]
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TransCon hGH CT-302
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Universal Trial Number (UTN)
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Trial acronym
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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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0
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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 at a starting dose of 0.24 mg/kg/week
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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 Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
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Assessment method [1]
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Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment
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Timepoint [1]
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26 weeks
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Secondary outcome [1]
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Annualized Height Velocity (AHV) at 26 Weeks of Weekly Lonapegsomatropin Treatment
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Assessment method [1]
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Annualized height velocity (AHV) at 26 weeks of weekly lonapegsomatropin (TransCon hGH) treatment. The AHV at each visit was modeled using ANCOVA adjusting for baseline age, peak GH levels (log transformed) at diagnosis, delta average-parental height SDS, prior GH dose level (log transformed), and prior GH dose duration (log transformed) as covariates and gender as a factor. Subjects who did not take prior GH treatment were not included in the model.
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Timepoint [1]
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26 weeks
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Secondary outcome [2]
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Number of Subjects With IGF-1 Standard Deviation Score (SDS) in the Range of 0.0 to +2.0 at 26 Weeks of Weekly Lonapegsomatropin Treatment
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Assessment method [2]
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IGF-1 Standard Deviation Score (SDS) is the number of standard deviations above or below the mean Insulin-like Growth Factor 1 (IGF-1) level for age and sex. IGF-1 SDS was derived using the LMS method as ((IGF-1/M)\^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from Bidlingmaier et al. (2014). A Standard Deviation Score of 0 represents the population mean.
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Timepoint [2]
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26 weeks
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Secondary outcome [3]
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Change in Height Standard Deviation Scores (SDS) at 26 Weeks of Weekly Lonapegsomatropin Treatment
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Assessment method [3]
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Height Standard Deviation Score (SDS) is the number of standard deviations above or below the mean height for age and sex. Height SDS was derived using the LMS method as ((Height/M)\^L)-1)/(L x S), where M = median, S = generalized coefficient of variation, and L = power in the Box-Cox transformation, the M, S, L values were obtained from 2000 CDC growth charts for the United States. A Standard Deviation Score of 0 represents the population mean. A higher change from baseline in Height SDS indicates a better outcome. The height SDS change from baseline at each visit was modeled using ANCOVA adjusting for baseline age, peak GH levels (log transformed) at diagnosis, delta average-parental height SDS, prior GH dose level (log transformed), and prior GH dose duration (log transformed) as covariates and gender as a factor. Subjects who did not take prior GH treatment were not included in the model.
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Timepoint [3]
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Baseline and 26 weeks
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Secondary outcome [4]
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Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation
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Assessment method [4]
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Number of participants with treatment emergent anti-hGH antibodies over 26 weeks of weekly lonapegsomatropin (TransCon hGH) treatment. All samples were negative for anti-hGH neutralizing antibodies.
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Timepoint [4]
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26 weeks
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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
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Minimum age
6
Months
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Maximum age
17
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. 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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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
13/11/2017
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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
19/03/2019
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Sample size
Target
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Accrual to date
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Final
146
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Monash Children's Hospital - 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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United States of America
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State/province [2]
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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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United States of America
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Iowa
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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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Minnesota
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United States of America
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Mississippi
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United States of America
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New Hampshire
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United States of America
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New York
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United States of America
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Ohio
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United States of America
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Oklahoma
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United States of America
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Oregon
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United States of America
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Texas
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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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Virginia
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Country [15]
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Canada
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State/province [15]
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Alberta
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Country [16]
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New Zealand
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State/province [16]
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Auckland
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Ascendis Pharma Endocrinology Division A/S
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Address
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Ethics approval
Ethics application status
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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.
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Trial website
https://clinicaltrials.gov/study/NCT03305016
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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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Aimee D Shu, MD
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Address
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Ascendis Pharma, Inc.
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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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Fax
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Email
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/16/NCT03305016/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/16/NCT03305016/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT03305016
Download to PDF