Growth Hormone Deficiency, PediatricPhase 3NCT02781727
A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)
A Phase 3 study testing Once weekly subcutaneous injection of TransCon hGH and Once daily subcutaneous injection of Genotropin in people with Growth Hormone Deficiency, Pediatric and hGH (Human Growth Hormone), run by Ascendis Pharma Endocrinology Division A/S at 53 sites in 14 countries.
Trial summary
A 52 week trial of TransCon hGH, a long-acting growth hormone product, versus human growth hormone therapy. TransCon hGH will be given once-a-week, human growth hormone (hGH) will be given daily. Approximately 150 prepubertal, hGH-treatment naïve children (males and females) with GHD will be included. Read more about this study
- Who is it for?
- Ages 3 to 12 years
- Conditions the study is for
- Growth Hormone Deficiency, Pediatric, hGH (Human Growth Hormone), Endocrine System Diseases
- What is being tested?
- Once weekly subcutaneous injection of TransCon hGH and Once daily subcutaneous injection of Genotropin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 162 participants
- Where
- 53 sitesin 14 countries
- Study dates
- 13 Dec 2016to 17 Jan 2019
- 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 3 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Prepubertal children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage 1 (Tanner 1982) aged:
- Boys: 3-12 years, inclusive
- Girls: 3-11 years, inclusive
- Impaired height (HT) defined as at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS ≤ -2.0) according to the 2000 CDC Growth Charts for the United States Methods and Development, available at http://www.cdc.gov/growthcharts/
- Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH level of ≤10 ng/mL, determined with a validated assay
- Bone age (BA) at least 6 months less than chronological age
- Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1)
- Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject (if the subject is able to read, understand, and sign)
Who cannot join
- Children with a body weight below 12 kg
- Prior exposure to recombinant hGH or IGF-1 therapy
- Children with past or present intracranial tumor growth as confirmed by a sellar MRI scan (with contrast) at Screening (MRI results from up to 6 months prior to Screening may be accepted)
- Children with psychosocial dwarfism
- Children with idiopathic short stature
- History or presence of malignant disease; any evidence of present tumor growth
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- Closed epiphyses
- Major medical conditions and/or presence of contraindication to hGH treatment
- Participation in any other trial of an investigational agent within 3 months prior to Screening
Trial locations and contacts
53 sites in 14 countries.
53 sites in 14 countries
United States16 sites
Ascendis Pharma Investigational Site
Birmingham, Alabama, 35233
Ascendis Pharma Investigational Site
Centennial, Colorado, 80112
Ascendis Pharma Investigational Site
Dallas, Texas, 75235
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
Little Rock, Arkansas, 72202
Ascendis Pharma Investigational Site
Los Angeles, California, 90048
Ascendis Pharma Investigational Site
Mineola, New York, 11501
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, 97239
Ascendis Pharma Investigational Site
Saint Paul, Minnesota, 55102
Ascendis Pharma Investigational Site
Tacoma, Washington, 98405
Russia16 sites
Ascendis Pharma Investigational Site
Izhevsk, 426009
Ascendis Pharma Investigational Site
Kazan', 420138
Ascendis Pharma Investigational Site
Krasnoyarsk, 620022
Ascendis Pharma Investigational Site
Moscow, 125373
Ascendis Pharma Investigational Site
Moscow, 127994
Ascendis Pharma Investigational Site
Nizhny Novgorod, 603136
Ascendis Pharma Investigational Site
Novosibirsk, 630048
Ascendis Pharma Investigational Site
Omsk, 644001
Ascendis Pharma Investigational Site
Saint Petersburg, 191144
Ascendis Pharma Investigational Site
Saint Petersburg, 194100
Ascendis Pharma Investigational Site
Samara, 443079
Ascendis Pharma Investigational Site
Saratov, 410054
Ascendis Pharma Investigational Site
Tomsk, 634050
Ascendis Pharma Investigational Site
Ufa, 450008
Ascendis Pharma Investigational Site
Vologda, 160022
Ascendis Pharma Investigational Site
Voronezh, 394024
Ukraine4 sites
Ascendis Pharma Investigational Site
Kharkiv, 61093
Ascendis Pharma Investigational Site
Kyiv, 01021
Ascendis Pharma Investigational Site
Kyiv, 04114
Ascendis Pharma Investigational Site
Odesa, 65031
Georgia3 sites
Ascendis Pharma Investigational Site
Tbilisi, 0144
Ascendis Pharma Investigational Site
Tbilisi, 0159
Ascendis Pharma Investigational Site
Tbilisi, 0162
Turkey (Türkiye)3 sites
Ascendis Pharma Investigational Site
Izmir, 35100
Ascendis Pharma Investigational Site
Melikgazi, 38039
Ascendis Pharma Investigational Site
Trabzon, 61080
Italy2 sites
Ascendis Pharma Investigational Site
Milan, 20157
Ascendis Pharma Investigational Site
Roma, 00165
Poland2 sites
Ascendis Pharma Investigatonal Site
Gdansk, 80-952
Ascendis Pharma Investigational Site
Warsaw, 02-691
About this study
Official title: A Multicenter, Phase 3, Randomized, Open-label, Active-controlled, Parallel-group Trial Investigating the Safety, Tolerability, and Efficacy of TransCon hGH Administered Once a Week Versus Standard Daily hGH Replacement Therapy Over 52 Weeks in Prepubertal Children With Growth Hormone Deficiency (GHD)
A 52 week trial of TransCon hGH, a long-acting growth hormone product, versus human growth hormone therapy. TransCon hGH will be given once-a-week, human growth hormone (hGH) will be given daily. Approximately 150 prepubertal, hGH-treatment naïve children (males and females) with GHD will be included. Randomization will occur in a 2:1 ratio (TransCon hGH : Genotropin). This is a global trial that will be conducted in Armenia, Australia, Belarus, Bulgaria, Georgia, Greece, Italy, New Zealand, Poland, Romania, Russia, Turkey, Ukraine, and the United States.
- Conditions
- Growth Hormone Deficiency, Pediatric, hGH (Human Growth Hormone), Endocrine System Diseases, Hormones, Pituitary Diseases
- Being tested
- Once weekly subcutaneous injection of TransCon hGH, Once daily subcutaneous injection of Genotropin
- 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, Sustained Release Growth Hormone, Growth Hormone Deficiency
- 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.
Once weekly subcutaneous injection of TransCon hGHDrug
Once weekly subcutaneous injection
Once daily subcutaneous injection of GenotropinDrug
Once daily subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| TransCon hGH | Experimental | Once weekly subcutaneous injection of TransCon hGH Receives: Drug: Once weekly subcutaneous injection of TransCon hGH |
| human growth hormone (Genotropin) | Active comparator | Once daily subcutaneous injection of Genotropin Receives: Drug: Once daily subcutaneous injection of Genotropin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Annualized height velocity (AHV) at 52 weeks for weekly lonapegsomatropin (TransCon hGH) and daily hGH treatment groups
Measured: 52 weeks
Secondary outcomes (5)
Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
Number of participants with Treatment-Emergent Adverse Events for the weekly lonapegsomatropin and daily hGH treatment groups
Measured: 52 Weeks
Annualized Height Velocity Over 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups
Annualized height velocity (AHV) over 52 weeks for weekly lonapegsomatropin and daily hGH treatment groups. AHV by visit was determined by ANCOVA model with multiple imputation. For each imputed data set, an ANCOVA model with by visit AHV as the dependent variable, treatment and gender as factors, baseline age, baseline peak GH levels (log transformed) at stimulation test, and baseline height SDS - average parental height SDS as covariates were fitted.
Measured: Week 5, Week 13, Week 26, Week 39 and Week 52
Change in Height Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups
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 Standard Deviation Score (SDS) indicates a better outcome. The change from baseline in height SDS by visit was determined by ANCOVA model and included baseline age, peak GH levels (log transformed) at stimulation test and baseline height SDS as covariates, as well as treatment and gender as factors.
Measured: Week 5, Week 13, Week 26, Week 39 and Week 52
Average IGF-1 Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups
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. Average IGF-1 SDS by visit was determined by ANCOVA. The ANCOVA model included baseline age, peak GH levels (log transformed) at stimulation test, baseline IGF-1 SDS as covariates, as well as treatment and gender as factors. Modeled values begin at Week 13 corresponding with achievement of IGF-1 steady state. Average IGF-1 SDS values by visit for the Lonapegsomatropin group were derived from a population pharmacodynamic model; the average IGF-1 SDS values for the Genotropin group are represented by observed values.
Measured: Week 13, Week 26, Week 39, and Week 52
Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation
Number of participants with treatment emergent anti-hGH binding antibody formation during the 52 week study. All samples were negative for anti-hGH neutralizing antibodies.
Measured: Start of study treatment through Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2016 (estimated)
- Study start13 Dec 2016
- Primary completion17 Jan 2019
- Study completion17 Jan 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.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 162 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ascendis Pharma Endocrinology Division A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, MD, Study director, Ascendis Pharma A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02781727
- Sponsor's protocol ID
- TransCon hGH CT-301
- Other IDs
- 2016-001145-11, 2016
- Status last verified
- Jun 2026
- First submitted
- 19 May 2016
- Last updated
- 7 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Thornton PS, Maniatis AK, Aghajanova E, Chertok E, Vlachopapadopoulou E, Lin Z, Song W, Christoffersen ED, Breinholt VM, Kovalenko T, Giorgadze E, Korpal-Szczyrska M, Hofman PL, Karpf DB, Shu AD, Beckert M. Weekly Lonapegsomatropin in Treatment-Naive Children With Growth Hormone Deficiency: The Phase 3 heiGHt Trial. J Clin Endocrinol Metab. 2021 Oct 21;106(11):3184-3195. doi: 10.1210/clinem/dgab529.
PubMed 34272849