A Phase 3 interventional study of Once weekly subcutaneous injection of TransCon hGH and Once daily subcutaneous injection of Genotropin in Growth Hormone Deficiency, Pediatric, hGH (Human Growth Hormone) and Endocrine System Diseases, sponsored by Ascendis Pharma Endocrinology Division A/S. Completed at 53 sites in 14 countries. Open to participants aged 3 Years to 12 Years. Per ClinicalTrials.gov, last updated 2026-07-07.
Sponsored by Ascendis Pharma Endocrinology Division A/S · Phase 3, Interventional, and Treatment
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.
252 studies on the registry are indexed under Dwarfism, Pituitary; 30 are open to participants now.
This study's enrollment of 162 is above the median of 60 across 169 interventional studies indexed under Dwarfism, Pituitary.
Browse Dwarfism, Pituitary studies →Ascendis Pharma Endocrinology Division A/S is the lead sponsor of 7 studies on the registry; 2 are open to participants now.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Prepubertal children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage 1 (Tanner 1982) aged:
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Once weekly subcutaneous injection of TransCon hGH
Drug: Once weekly subcutaneous injection of TransCon hGH
Once daily subcutaneous injection of Genotropin
Drug: Once daily subcutaneous injection of Genotropin
Once weekly subcutaneous injection
Once daily subcutaneous injection
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
Time frame: 52 weeks
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
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Start of study treatment through Week 52
| Milestone | Lonapegsomatropin | Daily hGH |
|---|---|---|
| Started | 106 | 56 |
| Completed | 105 | 56 |
| Not completed | 1 | 0 |
| Withdrew: Withdrawal by subject | 1 | 0 |
| Milestone | Lonapegsomatropin | Daily hGH |
|---|---|---|
| Started | 105 | 56 |
| Completed | 104 | 55 |
| Not completed | 1 | 1 |
| Withdrew: Withdrawal by subject | 1 | 0 |
| Withdrew: Lost to follow-up | 0 | 1 |
Annualized height velocity (AHV) at 52 weeks for weekly lonapegsomatropin (TransCon hGH) and daily hGH treatment groups
| cm/year | Lonapegsomatropin | Daily hGH |
|---|---|---|
| Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups | 11.17 ± 0.23 | 10.31 ± 0.30 |
Number of participants with Treatment-Emergent Adverse Events for the weekly lonapegsomatropin and daily hGH treatment groups
| Participants | Lonapegsomatropin | Daily hGH |
|---|---|---|
| TEAEs | 81 | 39 |
| TEAEs related to study drug | 12 | 10 |
| SAEs | 1 | 1 |
| SAEs related to study drug | 0 | 0 |
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.
| cm/year | Lonapegsomatropin | Daily hGH |
|---|---|---|
| Week 5 | 13.54 ± 1.07 | 12.83 ± 1.37 |
| Week 13 | 13.28 ± 0.49 | 12.22 ± 0.63 |
| Week 26 | 12.65 ± 0.32 | 11.21 ± 0.42 |
| Week 39 | 11.89 ± 0.26 | 10.90 ± 0.33 |
| Week 52 | 11.17 ± 0.23 | 10.31 ± 0.30 |
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.
| standard deviation score | Lonapegsomatropin | Daily hGH |
|---|---|---|
| Week 5 | 0.13 ± 0.02 | 0.12 ± 0.02 |
| Week 13 | 0.38 ± 0.02 | 0.33 ± 0.03 |
| Week 26 | 0.68 ± 0.03 | 0.58 ± 0.04 |
| Week 39 | 0.92 ± 0.03 | 0.80 ± 0.04 |
| Week 52 | 1.10 ± 0.04 | 0.96 ± 0.05 |
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.
| standard deviation score | Lonapegsomatropin | Daily hGH |
|---|---|---|
| Week 13 | 0.31 ± 0.09 | -0.60 ± 0.11 |
| Week 26 | 0.46 ± 0.08 | -0.51 ± 0.10 |
| Week 39 | 0.59 ± 0.09 | -0.30 ± 0.11 |
| Week 52 | 0.72 ± 0.09 | -0.02 ± 0.12 |
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.
| Participants | Lonapegsomatropin | Daily hGH |
|---|---|---|
| Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation | 6 | 2 |
Collected over From the first trial-related activity after the subject signed the informed consent until the end of the post-treatment follow-up period (up to week 52). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Lonapegsomatropin | 0/105 (0%) | 1/105 (1%) | 81/105 (77.1%) |
| Daily hGH | 0/56 (0%) | 1/56 (1.8%) | 39/56 (69.6%) |
| Event | Lonapegsomatropin | Daily hGH |
|---|---|---|
| ConcussionInjury, poisoning and procedural complications | 0/105 | 1/56 |
| AppendicitisInfections and infestations | 1/105 | 0/56 |
| Event | Lonapegsomatropin | Daily hGH |
|---|---|---|
| PharyngitisInfections and infestations | 10/105 | 10/56 |
| PyrexiaGeneral disorders | 16/105 | 5/56 |
| NasopharyngitisInfections and infestations | 12/105 | 8/56 |
| HeadacheNervous system disorders | 13/105 | 7/56 |
| CoughRespiratory, thoracic and mediastinal disorders | 10/105 | 4/56 |
| Upper respiratory tract infectionInfections and infestations | 6/105 | 5/56 |
| VomitingGastrointestinal disorders | 9/105 | 3/56 |
| Respiratory tract infectionInfections and infestations | 7/105 | 3/56 |
| Secondary hypothyroidismEndocrine disorders | 7/105 | 3/56 |
| DiarrheaGastrointestinal disorders | 6/105 | 3/56 |
The Intention-to-Treat (ITT) population included all randomized subjects who had received at least 1 dose of active treatment.
| Age, Continuous(years) | Lonapegsomatropin | Daily hGH | Total |
|---|---|---|---|
| Mean | 8.5 ± 2.7 | 8.5 ± 2.8 | 8.5 ± 2.7 |
| Age, Customized(Participants) | Lonapegsomatropin | Daily hGH | Total |
|---|---|---|---|
| Age, Categorical — <6 years | 25 | 14 | 39 |
| Age, Categorical — ≥6 years | 80 | 42 | 122 |
| Sex: Female, Male(Participants) | Lonapegsomatropin | Daily hGH | Total |
|---|---|---|---|
| Female | 19 | 10 | 29 |
| Male | 86 | 46 | 132 |
| Race/Ethnicity, Customized(Participants) | Lonapegsomatropin | Daily hGH | Total |
|---|---|---|---|
| Race — Asian | 1 | 0 | 1 |
| Race — Black or African American | 2 | 1 | 3 |
| Race — White | 100 | 52 | 152 |
| Race — Other | 2 | 3 | 5 |
| Race/Ethnicity, Customized(Participants) | Lonapegsomatropin | Daily hGH | Total |
|---|---|---|---|
| Ethnicity — Hispanic or Latino | 5 | 2 | 7 |
| Ethnicity — Not Hispanic or Latino | 100 | 54 | 154 |
| Height(cm) | Lonapegsomatropin | Daily hGH | Total |
|---|---|---|---|
| Mean | 112.93 ± 14.09 | 112.15 ± 15.29 | 112.66 ± 14.48 |
| Height SDS(standard deviation score) | Lonapegsomatropin | Daily hGH | Total |
|---|---|---|---|
| Mean | -2.89 ± 0.85 | -3.00 ± 0.90 | -2.93 ± 0.87 |
| Weight(kg) | Lonapegsomatropin | Daily hGH | Total |
|---|---|---|---|
| Mean | 21.01 ± 6.54 | 21.20 ± 6.67 | 21.08 ± 6.56 |
3 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Dec 2016 to primary completion in Jan 2019, 25 months.
Results were first posted in Jan 2022, 2 years 11 months after primary completion. A results delay was requested in Dec 2019.
Of 19 phase 3 interventional studies under Dwarfism, Pituitary, half reached primary completion within 24 months of starting (the median), counting those still running; a quarter within 18 and three quarters within 30. This study's 25 months is close to that.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Ascendis Pharma Endocrinology Division A/S