A Phase 3 interventional study of TransCon hGH in Growth Hormone Deficiency, Pediatric, Endocrine System Diseases and Hormone Deficiency, sponsored by Ascendis Pharma Endocrinology Division A/S. Completed at 24 sites in 4 countries. Open to participants aged 6 Months to 17 Years. Per ClinicalTrials.gov, last updated 2026-07-07.
Sponsored by Ascendis Pharma Endocrinology Division A/S · Phase 3, Interventional, and Treatment
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.
252 studies on the registry are indexed under Dwarfism, Pituitary; 30 are open to participants now.
This study's enrollment of 146 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.
Counted across the registry records on this site, refreshed daily.
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.
6 months to 17 years old, inclusive, at Visit 1
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.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
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.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
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: TransCon hGH
Once weekly subcutaneous injection at a starting dose of 0.24 mg/kg/week
Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]
Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment
Time frame: 26 weeks
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.
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
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.
Time frame: 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.
Time frame: 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.
Time frame: 26 weeks
| Milestone | Lonapegsomatropin |
|---|---|
| Started | 146 |
| Completed | 144 |
| Not completed | 2 |
| Withdrew: Withdrawal by subject | 2 |
Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment
| Participants | Lonapegsomatropin |
|---|---|
| TEAE | 83 |
| Related TEAE | 6 |
| Serious TEAE | 1 |
| Related Serious TEAE | 0 |
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.
| cm/year | Lonapegsomatropin |
|---|---|
| Annualized Height Velocity (AHV) at 26 Weeks of Weekly Lonapegsomatropin Treatment | 8.72 ± 0.24 |
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.
| Participants | Lonapegsomatropin |
|---|---|
| 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 | 74 |
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.
| standard deviation score | Lonapegsomatropin |
|---|---|
| Change in Height Standard Deviation Scores (SDS) at 26 Weeks of Weekly Lonapegsomatropin Treatment | 0.25 ± 0.02 |
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.
| Participants | Lonapegsomatropin |
|---|---|
| Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation | 4 |
Collected over 26 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Lonapegsomatropin | 0/146 (0%) | 1/146 (0.7%) | 65/146 (44.5%) |
| Event | Lonapegsomatropin |
|---|---|
| Atrioventricular blockCardiac disorders | 1/146 |
| Chest painGeneral disorders | 1/146 |
| Event | Lonapegsomatropin |
|---|---|
| PyrexiaGeneral disorders | 17/146 |
| NasopharyngitisInfections and infestations | 14/146 |
| Upper respiratory tract infectionInfections and infestations | 14/146 |
| HeadacheNervous system disorders | 12/146 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 8/146 |
| Age, Continuous(years) | Lonapegsomatropin |
|---|---|
| Mean | 10.6 ± 3.9 |
| Age, Customized(Participants) | Lonapegsomatropin |
|---|---|
| Age, Categorical: — <3 years | 4 |
| Age, Categorical: — ≥3 and <6 years | 20 |
| Age, Categorical: — ≥6 to <11 years (girls) or ≥6 to <12 years (boys) | 55 |
| Age, Categorical: — ≥11 years (girls) or ≥12 years (boys) | 67 |
| Sex: Female, Male(Participants) | Lonapegsomatropin |
|---|---|
| Female | 36 |
| Male | 110 |
| Ethnicity (NIH/OMB)(Participants) | Lonapegsomatropin |
|---|---|
| Hispanic or Latino | 10 |
| Not Hispanic or Latino | 124 |
| Unknown or Not Reported | 12 |
| Race/Ethnicity, Customized(Participants) | Lonapegsomatropin |
|---|---|
| Race — Asian | 6 |
| Race — Black or African American | 3 |
| Race — Native Hawaiian or Other Pacific Islander | 2 |
| Race — White | 124 |
| Race — More than one race | 2 |
| Race — Unknown | 9 |
| Height(cm) | Lonapegsomatropin |
|---|---|
| Mean | 132.4 ± 22.5 |
| Height SDS(standard deviation score) | Lonapegsomatropin |
|---|---|
| Mean | -1.42 ± 0.84 |
| Weight(kg) | Lonapegsomatropin |
|---|---|
| Mean | 32.3 ± 14.2 |
2 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Nov 2017 to primary completion in Mar 2019, 16 months.
Results were first posted in Jan 2022, 2 years 9 months after primary completion. A results delay was requested in Feb 2020.
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 16 months is below 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