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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-001145-11 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TransCon hGH | Experimental | Once weekly subcutaneous injection of TransCon hGH |
|
| human growth hormone (Genotropin) | Active Comparator | Once daily subcutaneous injection of Genotropin |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Once weekly subcutaneous injection of TransCon hGH | Drug | Once weekly subcutaneous injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 52 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 52 Weeks |
| Annualized Height Velocity Over 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups |
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Inclusion Criteria:
Prepubertal children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage 1 (Tanner 1982) aged:
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)
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director, MD | Ascendis Pharma A/S | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ascendis Pharma Investigational Site | Birmingham | Alabama | 35233 | United States | ||
| Ascendis Pharma Investigational Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34272849 | Derived | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Lonapegsomatropin | Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH) |
| FG001 | Daily hGH | Once daily subcutaneous injection of Genotropin |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Pre-dosing Period |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 12, 2017 | Sep 24, 2021 |
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| Once daily subcutaneous injection of Genotropin | Drug | Once daily subcutaneous injection |
|
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. |
| 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. | 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. | 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. | Start of study treatment through Week 52 |
| Little Rock |
| Arkansas |
| 72202 |
| United States |
| Ascendis Pharma Investigational Site | Los Angeles | California | 90048 | United States |
| Ascendis Pharma Investigational Site | Orange | California | 92868 | United States |
| Ascendis Pharma Investigational Site | Centennial | Colorado | 80112 | United States |
| Ascendis Pharma Investigational Site | Jacksonville | Florida | 32207 | United States |
| Ascendis Pharma Investigational Site | Orlando | Florida | 32806 | United States |
| Ascendis Pharma Investigational Site | Saint Paul | Minnesota | 55102 | United States |
| Ascendis Pharma Investigational Site | Jackson | Mississippi | 39216 | United States |
| Ascendis Pharma Investigational Site | Lebanon | New Hampshire | 03756 | United States |
| Ascendis Pharma Investigational Site | Mineola | New York | 11501 | United States |
| Ascendis Pharma Investigational Site | Oklahoma City | Oklahoma | 73104 | United States |
| Ascendis Pharma Investigational Site | Portland | Oregon | 97239 | United States |
| Ascendis Pharma Investigational Site | Dallas | Texas | 75235 | United States |
| Ascendis Pharma Investigational Site | Fort Worth | Texas | 76104 | United States |
| Ascendis Pharma Investigational Site | Tacoma | Washington | 98405 | United States |
| Ascendis Pharma Investigational Site | Yerevan | 0075 | Armenia |
| Ascendis Pharma Investigational Site | Clayton | 3168 | Australia |
| Ascendis Pharma Investigational Site | Minsk | 220020 | Belarus |
| Ascendis Pharma Investigational Site | Varna | 9010 | Bulgaria |
| Ascendis Pharma Investigational Site | Tbilisi | 0144 | Georgia |
| Ascendis Pharma Investigational Site | Tbilisi | 0159 | Georgia |
| Ascendis Pharma Investigational Site | Tbilisi | 0162 | Georgia |
| Ascendis Pharma Investigational Site | Athens | 11527 | Greece |
| Ascendis Pharma Investigational Site | Milan | 20157 | Italy |
| Ascendis Pharma Investigational Site | Roma | 00165 | Italy |
| Ascendis Pharma Investigational Site | Grafton | 1023 | New Zealand |
| Ascendis Pharma Investigatonal Site | Gdansk | 80-952 | Poland |
| Ascendis Pharma Investigational Site | Warsaw | 02-691 | Poland |
| Ascendis Pharma Investigational Site | Iași | 700111 | Romania |
| Ascendis Pharma Investigational Site | Izhevsk | 426009 | Russia |
| Ascendis Pharma Investigational Site | Kazan' | 420138 | Russia |
| Ascendis Pharma Investigational Site | Krasnoyarsk | 620022 | Russia |
| Ascendis Pharma Investigational Site | Moscow | 125373 | Russia |
| Ascendis Pharma Investigational Site | Moscow | 127994 | Russia |
| Ascendis Pharma Investigational Site | Nizhny Novgorod | 603136 | Russia |
| Ascendis Pharma Investigational Site | Novosibirsk | 630048 | Russia |
| Ascendis Pharma Investigational Site | Omsk | 644001 | Russia |
| Ascendis Pharma Investigational Site | Saint Petersburg | 191144 | Russia |
| Ascendis Pharma Investigational Site | Saint Petersburg | 194100 | Russia |
| Ascendis Pharma Investigational Site | Samara | 443079 | Russia |
| Ascendis Pharma Investigational Site | Saratov | 410054 | Russia |
| Ascendis Pharma Investigational Site | Tomsk | 634050 | Russia |
| Ascendis Pharma Investigational Site | Ufa | 450008 | Russia |
| Ascendis Pharma Investigational Site | Vologda | 160022 | Russia |
| Ascendis Pharma Investigational Site | Voronezh | 394024 | Russia |
| Ascendis Pharma Investigational Site | Izmir | 35100 | Turkey (Türkiye) |
| Ascendis Pharma Investigational Site | Melikgazi | 38039 | Turkey (Türkiye) |
| Ascendis Pharma Investigational Site | Trabzon | 61080 | Turkey (Türkiye) |
| Ascendis Pharma Investigational Site | Kharkiv | 61093 | Ukraine |
| Ascendis Pharma Investigational Site | Kyiv | 01021 | Ukraine |
| Ascendis Pharma Investigational Site | Kyiv | 04114 | Ukraine |
| Ascendis Pharma Investigational Site | Odesa | 65031 | Ukraine |
| COMPLETED |
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| NOT COMPLETED |
|
|
| Treatment Period |
|
|
The Intention-to-Treat (ITT) population included all randomized subjects who had received at least 1 dose of active treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | Lonapegsomatropin | Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH) |
| BG001 | Daily hGH | Once daily subcutaneous injection of Genotropin |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Height | Mean | Standard Deviation | cm |
| |||||||||||||||
| Height SDS | 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. | Mean | Standard Deviation | standard deviation score |
| ||||||||||||||
| Weight | Mean | Standard Deviation | kg |
| |||||||||||||||
| Body Mass Index (BMI) | Mean | Standard Deviation | kg/m^2 |
| |||||||||||||||
| BMI SDS | BMI Standard Deviation Score (SDS) is the number of standard deviations above or below the mean body mass index for age and sex. BMI SDS was derived using the LMS method as ((BMI/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. | Mean | Standard Deviation | standard deviation score |
| ||||||||||||||
| IGF-1 SDS | 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. | Mean | Standard Deviation | standard deviation score |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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 | The Intention-to-Treat (ITT) population included all randomized subjects who had received at least 1 dose of active treatment. | Posted | Least Squares Mean | Standard Error | cm/year | 52 weeks |
|
|
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| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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 | The safety analysis population included all randomized subjects who had received at least 1 dose of active treatment. | Posted | Count of Participants | Participants | 52 Weeks |
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| Secondary | 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. | The Intention-to-Treat (ITT) population included all randomized subjects who had received at least 1 dose of active treatment. | Posted | Least Squares Mean | Standard Error | cm/year | Week 5, Week 13, Week 26, Week 39 and Week 52 |
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| Secondary | 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. | The Intention-to-Treat (ITT) population included all randomized subjects who had received at least 1 dose of active treatment. | Posted | Least Squares Mean | Standard Error | standard deviation score | Week 5, Week 13, Week 26, Week 39 and Week 52 |
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| Secondary | 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. | The Intention-to-Treat (ITT) population included all randomized subjects who had received at least 1 dose of active treatment. | Posted | Least Squares Mean | Standard Error | standard deviation score | Week 13, Week 26, Week 39, and Week 52 |
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| Secondary | 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. | The safety analysis population included all randomized subjects who had received at least 1 dose of active treatment. | Posted | Count of Participants | Participants | Start of study treatment through Week 52 |
|
|
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)
All adverse events (AEs) were collected in response to a general question about the subject's well-being and any possible changes from the previous visit, but were not specifically solicited. AEs, including any serious adverse events, were collected through the end of trial (ie, the Week 52 Visit 6). AEs ongoing at Visit 6 or the time of premature trial discontinuation were followed until the event was resolved or deemed stable by the investigator.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Lonapegsomatropin | Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH) | 0 | 105 | 1 | 105 | 81 | 105 |
| EG001 | Daily hGH | Once daily subcutaneous injection of Genotropin | 0 | 56 | 1 | 56 | 39 | 56 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Appendicitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Concussion | Injury, poisoning and procedural complications | MedDRA 19.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 19.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Diarrhea | Gastrointestinal disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 19.0 | Systematic Assessment |
| |
| Secondary hypothyroidism | Endocrine disorders | MedDRA 19.0 | Systematic Assessment |
|
Not provided
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Ascendis Pharma | +45 61161658 | Asnd_registryinquiries@ascendispharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 19, 2019 | Sep 24, 2021 | SAP_001.pdf |
| ID | Term |
|---|---|
| D004393 | Dwarfism, Pituitary |
| D004700 | Endocrine System Diseases |
| D010900 | Pituitary Diseases |
| D005183 | Failure to Thrive |
| ID | Term |
|---|---|
| D004392 | Dwarfism |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D001849 | Bone Diseases, Endocrine |
| D007018 | Hypopituitarism |
| D007027 | Hypothalamic Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ≥6 years |
|
| Male |
|
| Black or African American |
|
| White |
|
| Other |
|
| Not Hispanic or Latino |
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
Once daily subcutaneous injection of Genotropin
|
|
|