Growth Hormone Deficiency, PediatricPhase 3enliGHtenNCT03344458
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
A Phase 3 study testing TransCon hGH in people with Growth Hormone Deficiency, Pediatric and Endocrine System Diseases, run by Ascendis Pharma A/S at 42 sites in 11 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 18 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
- 298 participants
- Where
- 42 sitesin 11 countries
- Study dates
- 19 Dec 2017to 21 Feb 2023
- Phase
- Phase 3
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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Children who have completed a prior phase 3 TransCon hGH trial
- Children who have not permanently discontinued study drug in the prior trial
- 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
- Poorly-controlled diabetes mellitus (HbA1c ≥ 8.0%) or diabetic complications
- Evidence of closed epiphyses, defined as bone age > 14.0 years for females or > 16.0 years for males
- Major medical conditions unless approved by Medical Expert
- Known hypersensitivity to the components of the trial medication
- Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver)
- Pregnancy
- Any other reason that in the opinion of the investigator would prevent the subject from completing participation or following the trial schedule
Trial locations and contacts
42 sites in 11 countries.
42 sites in 11 countries
United States22 sites
Ascendis Pharma Investigational Site
Birmingham, Alabama, 35233
Ascendis Pharma Investigational Site
Centennial, Colorado, 80112
Ascendis Pharma Investigational Site
Charlottesville, Virginia, 22908
Ascendis Pharma Investigational Site
Cleveland, Ohio, 44195
Ascendis Pharma Investigational Site
Dallas, Texas, 75390
Ascendis Pharma Investigational Site
Fort Worth, Texas, 76104
Ascendis Pharma Investigational Site
Jackson, Mississippi, 39216
Ascendis Pharma Investigational Site
Jacksonville, Florida, 32207
Ascendis Pharma Investigational Site
Lebanon, New Hampshire, 03756
Ascendis Pharma Investigational Site
Los Angeles, California, 90048
Ascendis Pharma Investigational Site
Mineola, New York, 11501
Ascendis Pharma Investigational Site
New York, New York, 10029
Ascendis Pharma Investigational Site
Norfolk, Virginia, 23507
Ascendis Pharma Investigational Site
Oklahoma City, Oklahoma, 73104
Ascendis Pharma Investigational Site
Orange, California, 92868
Ascendis Pharma Investigational Site
Orlando, Florida, 32806
Ascendis Pharma Investigational Site
Portland, Oregon, 97227
Ascendis Pharma Investigational Site
Portland, Oregon, 97239
Ascendis Pharma Investigational Site
Sacramento, California, 95821
Ascendis Pharma Investigational Site
Saint Paul, Minnesota, 55102
Ascendis Pharma Investigational Site
Tacoma, Washington, 98405
Ascendis Pharma Investigational Site
Tallahassee, Florida, 32308
Russia8 sites
Ascendis Pharma Investigational Site
Izhevsk, 426009
Ascendis Pharma Investigational Site
Krasnoyarsk, 660022
Ascendis Pharma Investigational Site
Moscow, 117036
Ascendis Pharma Investigational Site
Saint Petersburg, 194100
Ascendis Pharma Investigational Site
Saratov, 410054
Ascendis Pharma Investigational Site
Ufa, 450008
Ascendis Pharma Investigational Site
Vologda, 160022
Ascendis Pharma Investigational Site
Voronezh, 394024
Ukraine3 sites
Ascendis Pharma Investigational Site
Kharkiv, 61093
Ascendis Pharma Investigational Site
Kyiv, 01021
Ascendis Pharma Investigational Site
Odesa, 65031
Poland2 sites
Ascendis Pharma Investigational Site
Gdansk, 80-952
Ascendis Pharma Investigational Site
Warsaw, 02-691
Australia1 site
Ascendis Pharma Investigational Site
Clayton, Victoria, 3168
About this study
Official 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
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.
- 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 of TransCon hGH
| 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 Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability]
Long-term safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment
Measured: Up to Week 208
Secondary outcomes (3)
Annualized Height Velocity by Visit
Annualized height velocity (AHV) by visit with long-term dosing of weekly lonapegsomatropin treatment
Measured: Up to Week 208
IGF-1 SDS by Visit
Insulin-like Growth Factor-1 (IGF-1) standard deviation score (SDS) by visit with long-term dosing of weekly lonapegsomatropin treatment at Week 13, Week 52, Week 104, Week 156, and Week 208. IGF-1 SDS is the number of standard deviations above or below the mean IGF-1 level for a child of the same age and sex. The target range for IGF-1 SDS was 0 to +2. An IGF-1 SDS of 0 represents the population mean. If the IGF-1 SDS is < 0, then the negative score indicates an IGF-1 below the mean IGF-1 for a child of the same age and sex. If the IGF-1 SDS is > 0, then the positive score indicates an IGF-1 above the mean IGF-1 for a child of the same age and sex. A positive score of IGF-1 SDS indicates a better outcome.
Measured: Up to Week 208
Height SDS - Change From Baseline
Change in Height Standard Deviation Score (SDS) from baseline with long-term dosing of weekly lonapegsomatropin treatment at Week 13, Week 52, Week 104, Week 156, and Week 208. Height SDS is the number of standard deviations above or below the mean height for age and sex. A Standard Deviation Score of 0 equals the population mean. If the Height SDS is < 0, then the negative score indicates a height below the mean height for a child of the same age and sex. If the Height SDS is > 0, then the positive score indicates a height above the mean height for a child of the same age and sex. A positive change from baseline in Height SDS indicates improved outcomes.
Measured: Up to Week 208
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2017
- Study start19 Dec 2017
- Primary completion21 Feb 2023
- Study completion21 Feb 2023
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.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- 298 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ascendis Pharma A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, Ascendis Pharma A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03344458
- Sponsor's protocol ID
- TransCon hGH CT-301EXT
- Other IDs
- 2017-003410-20, 2017
- Acronym
- enliGHten
- Status last verified
- Jun 2026
- First submitted
- 14 Nov 2017
- Last updated
- 7 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Maniatis AK, Casella SJ, Nadgir UM, Hofman PL, Saenger P, Chertock ED, Aghajanova EM, Korpal-Szczyrska M, Vlachopapadopoulou E, Malievskiy O, Chaychenko T, Cappa M, Song W, Mao M, Mygind PH, Smith AR, Chessler SD, Komirenko AS, Beckert M, Shu AD, Thornton PS. Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results. J Clin Endocrinol Metab. 2022 Jun 16;107(7):e2680-e2689. doi: 10.1210/clinem/dgac217.
PubMed 35428884