Growth Hormone Deficiency, PediatricPhase 3NCT03305016
A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone Deficiency
A Phase 3 study testing TransCon hGH in people with Growth Hormone Deficiency, Pediatric and Endocrine System Diseases, run by Ascendis Pharma Endocrinology Division A/S at 24 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 months to 17 years
- Conditions the study is for
- Growth Hormone Deficiency, Pediatric, Endocrine System Diseases, Hormone Deficiency
- What is being tested?
- TransCon hGH
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 146 participants
- Where
- 24 sitesin 4 countries
- Study dates
- 13 Nov 2017to 19 Mar 2019
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Investigator-determined GHD diagnosis prior to the historical initiation of daily hGH therapy.
- 6 months to 17 years old, inclusive, at Visit 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
- 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
- Tanner stage < 5 at Visit 1
- Open epiphyses (bone age ≤14.0 years for females or ≤16.0 years for males)
- 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
Who cannot join
- Weight of < 5.5 kg or > 80 kg at Visit 1
- Females of child-bearing potential
- History of malignant disease
- 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)
- Poorly-controlled diabetes mellitus (HbA1c >8.0%) or diabetic complications
- Known neutralizing antibodies against hGH
Show all 12Show fewer
- Major medical conditions, unless approved by Medical Monitor
- Pregnancy
- Presence of contraindications to hGH treatment
- Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver)
- Participation in any other trial of an investigational agent within 30 days prior to Visit 1
- Prior exposure to investigational hGH
Trial locations and contacts
24 sites in 4 countries.
24 sites in 4 countries
United States21 sites
University of Alabama
Birmingham, Alabama, 35233
Rocky Mountain Pediatric Endocrinology
Centennial, Colorado, 80112
University of Virginia Children's Hospital
Charlottesville, Virginia, 22903
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Children's Medical Center
Dallas, Texas, 75235
Cook Children's Medical Center
Fort Worth, Texas, 76104
University of Iowa
Iowa City, Iowa, 52242
University of Mississippi Medical Center
Jackson, Mississippi, 39216
Nemours Children's Health System
Jacksonville, Florida, 32207
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756
Neufeld Medical Group Inc.
Los Angeles, California, 90048
NYU Winthrop Hospital
Mineola, New York, 11501
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Orlando Health Inc.
Orlando, Florida, 32806
Children's Diabetes and Endocrine Center
Portland, Oregon, 97227
Oregon Health & Science University
Portland, Oregon, 97239
Center of Excellence in Diabetes and Endocrinology
Sacramento, California, 95821
Children's Minnesota
Saint Paul, Minnesota, 55102
Tallahassee Memorial Hospital
Tallahassee, Florida, 32308
New Zealand1 site
The Liggins Institute, The University of Auckland
Grafton, Auckland, 1023
About this study
Official 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
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.
- Conditions
- Growth Hormone Deficiency, Pediatric, Endocrine System Diseases, Hormone Deficiency, Pituitary Diseases
- Being tested
- TransCon hGH
- Keywords
- Human Growth Hormone, hGH, GHD, rhGH, Pediatric Growth Hormone Deficiency, Long Acting Growth Hormone, Somatropin, Prodrug, Growth Failure, Growth Hormone Replacement Therapy, Sustained Release Growth Hormone, Growth Hormone Deficiency, TransCon GH
- Medical subject headings
- Dwarfism, Pituitary, Endocrine System Diseases, Pituitary Diseases, Failure to Thrive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TransCon hGHDrug
Once weekly subcutaneous injection at a starting dose of 0.24 mg/kg/week
| Study group | Role | What they receive |
|---|---|---|
| TransCon hGH | Experimental | Once weekly subcutaneous injection of TransCon hGH Receives: Drug: TransCon hGH |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment
Measured: 26 weeks
Secondary outcomes (4)
Annualized Height Velocity (AHV) at 26 Weeks of Weekly Lonapegsomatropin Treatment
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.
Measured: 26 weeks
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
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.
Measured: 26 weeks
Change in Height Standard Deviation Scores (SDS) at 26 Weeks of Weekly Lonapegsomatropin Treatment
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.
Measured: Baseline and 26 weeks
Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation
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.
Measured: 26 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2017
- Study start13 Nov 2017
- Primary completion19 Mar 2019
- Study completion19 Mar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Growth Hormone Deficiency, PediatricStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- All study participants will receive TransCon hGH
- Masking
- None (open label)
- Enrolment
- 146 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ascendis Pharma Endocrinology Division A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Aimee Shu, MD, Study director, Ascendis Pharma, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03305016
- Sponsor's protocol ID
- TransCon hGH CT-302
- Other IDs
- U1111-1199-8218
- Status last verified
- Jun 2026
- First submitted
- 4 Oct 2017
- Last updated
- 7 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT03305016
- Acronym / sponsor ID
- TransCon hGH CT-302
- Conditions
- Growth Hormone Deficiency, Pediatric, Endocrine System Diseases, Hormone Deficiency, Pituitary Diseases
- Keywords
- Human Growth Hormone, hGH, GHD, rhGH, Pediatric Growth Hormone Deficiency, Long Acting Growth Hormone, Somatropin, Prodrug
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 146 participants (actual)
- Lead sponsor
- Ascendis Pharma Endocrinology Division A/SIndustry (pharma, biotech, devices)
- Study start
- 13 Nov 2017 (actual)
- Primary completion
- 19 Mar 2019 (actual)
- Study completion
- 19 Mar 2019 (actual)
- First posted
- 9 Oct 2017 (actual)
- Results
- Posted 4 Jan 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- All study participants will receive TransCon hGH
| Arm | Type | Receives |
|---|---|---|
| TransCon hGH | Experimental | Drug: TransCon hGHOnce weekly subcutaneous injection of TransCon hGH |
Who may qualify
- Ages
- 6 months to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Investigator-determined GHD diagnosis prior to the historical initiation of daily hGH therapy.
- 6 months to 17 years old, inclusive, at Visit 1
- Tanner stage < 5 at Visit 1
- Open epiphyses (bone age ≤14.0 years for females or ≤16.0 years for males)
- 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
Key exclusion criteria (12 listed)
- Weight of < 5.5 kg or > 80 kg at Visit 1
- Females of child-bearing potential
- History of malignant disease
- 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)
- Poorly-controlled diabetes mellitus (HbA1c >8.0%) or diabetic complications
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TransCon hGHOnce weekly subcutaneous injection at a starting dose of 0.24 mg/kg/week | Drug | TransCon hGH |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: 26 weeks
Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment
4 secondary outcomes
Annualized Height Velocity (AHV) at 26 Weeks of Weekly Lonapegsomatropin Treatment
Time frame: 26 weeks
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
Time frame: 26 weeks
Change in Height Standard Deviation Scores (SDS) at 26 Weeks of Weekly Lonapegsomatropin Treatment
Time frame: Baseline and 26 weeks
Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation
Time frame: 26 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Aimee Shu, MD study director
Ascendis Pharma, Inc.
24 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing TransCon hGH in people with Growth Hormone Deficiency, Pediatric and Endocrine System Diseases, run by Ascendis Pharma Endocrinology Division A/S at 24 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in United States, Australia, Canada, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Jan 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: 26 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jul 2026.