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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1199-8218 | Other Identifier | WHO UTN |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TransCon hGH | Experimental | Once weekly subcutaneous injection of TransCon hGH |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TransCon hGH | Drug | Once weekly subcutaneous injection at a starting dose of 0.24 mg/kg/week |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability] | Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment | 26 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Investigator-determined GHD diagnosis prior to the historical initiation of daily hGH therapy.
6 months to 17 years old, inclusive, at Visit 1
Tanner stage < 5 at Visit 1
Open epiphyses (bone age ≤14.0 years for females or ≤16.0 years for males)
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
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Aimee Shu, MD | Ascendis Pharma, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama | Birmingham | Alabama | 35233 | United States | ||
| Neufeld Medical Group Inc. |
A total of 162 subjects were screened for entry into this trial; of these, 16 subjects did not meet eligibility criteria and were considered screen failures.
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| ID | Title | Description |
|---|---|---|
| FG000 | Lonapegsomatropin | Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH) at a starting dose of 0.24 mg/kg/week |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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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 | Aug 29, 2017 | Sep 24, 2021 |
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All study participants will receive TransCon hGH
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| 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. | 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. | 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. | 26 weeks |
| Los Angeles |
| California |
| 90048 |
| United States |
| Center of Excellence in Diabetes and Endocrinology | Sacramento | California | 95821 | United States |
| Rocky Mountain Pediatric Endocrinology | Centennial | Colorado | 80112 | United States |
| Nemours Children's Health System | Jacksonville | Florida | 32207 | United States |
| Orlando Health Inc. | Orlando | Florida | 32806 | United States |
| Tallahassee Memorial Hospital | Tallahassee | Florida | 32308 | United States |
| University of Iowa | Iowa City | Iowa | 52242 | United States |
| Children's Minnesota | Saint Paul | Minnesota | 55102 | United States |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| Dartmouth Hitchcock Medical Center | Lebanon | New Hampshire | 03756 | United States |
| NYU Winthrop Hospital | Mineola | New York | 11501 | United States |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
| Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | United States |
| University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma | 73104 | United States |
| Children's Diabetes and Endocrine Center | Portland | Oregon | 97227 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Children's Medical Center | Dallas | Texas | 75235 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| University of Virginia Children's Hospital | Charlottesville | Virginia | 22903 | United States |
| Children's Hospital of The King's Daughters | Norfolk | Virginia | 23507 | United States |
| Monash Children's Hospital | Clayton | Victoria | 3168 | Australia |
| Stollery Children's Hospital | Edmonton | Alberta | T6G 2B7 | Canada |
| The Liggins Institute, The University of Auckland | Grafton | Auckland | 1023 | New Zealand |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Lonapegsomatropin | Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH) at a starting dose of 0.24 mg/kg/week |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Age, Customized | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| 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 |
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| Weight | Mean | Standard Deviation | kg |
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| Body Mass Index (BMI) | Mean | Standard Deviation | kg/m^2 |
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| 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 |
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| 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 | Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability] | Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment | The Full Analysis Set included all subjects who received at least 1 dose of trial drug during the trial and who had any follow-up data. | Posted | Count of Participants | Participants | 26 weeks |
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| Secondary | 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. | The Full Analysis Set included all subjects who received at least 1 dose of trial drug during the trial and who had any follow-up data. | Posted | Least Squares Mean | Standard Error | cm/year | 26 weeks |
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| Secondary | 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. | The Full Analysis Set included all subjects who received at least 1 dose of trial drug during the trial and who had any follow-up data. | Posted | Count of Participants | Participants | 26 weeks |
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| Secondary | 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. | The Full Analysis Set included all subjects who received at least 1 dose of trial drug during the trial and who had any follow-up data. | Posted | Least Squares Mean | Standard Error | standard deviation score | Baseline and 26 weeks |
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| Secondary | 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. | The Full Analysis Set included all subjects who received at least 1 dose of trial drug during the trial and who had any follow-up data. | Posted | Count of Participants | Participants | 26 weeks |
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26 weeks
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| 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) at a starting dose of 0.24 mg/kg/week | 0 | 146 | 1 | 146 | 65 | 146 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrioventricular block | Cardiac disorders | MedDRA (19.0) | Systematic Assessment |
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| Chest pain | General disorders | MedDRA (19.0) | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA (19.0) | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA (19.0) | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA (19.0) | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (19.0) | Systematic Assessment |
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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 | Mar 14, 2019 | Sep 24, 2021 | SAP_001.pdf |
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| 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 |
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| ≥6 to <11 years (girls) or ≥6 to <12 years (boys) |
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| ≥11 years (girls) or ≥12 years (boys) |
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| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| White |
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| More than one race |
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| Unknown |
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| Title | Measurements |
|---|---|
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| Related Serious TEAE |
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| Participants |
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