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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-003410-20 | EudraCT Number |
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A multicenter, phase 3, long-term extension trial of TransCon hGH administered once-weekly in children with growth hormone deficiency (GHD) who previously participated in a phase 3 TransCon hGH trial. Approximately 300 children (males and females) with 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, Poland, Bulgaria, Ukraine, Armenia, Russia and Australia.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TransCon hGH | Experimental | Once weekly subcutaneous injection of TransCon hGH |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TransCon hGH | Drug | Once weekly subcutaneous injection of TransCon hGH |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability] | Long-term safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment | Up to Week 208 |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Height Velocity by Visit | Annualized height velocity (AHV) by visit with long-term dosing of weekly lonapegsomatropin treatment | Up to Week 208 |
| IGF-1 SDS by Visit | Insulin-like Growth Factor-1 (IGF-1) standard deviation score (SDS) by visit with long-term dosing of weekly lonapegsomatropin treatment at Week 13, Week 52, Week 104, Week 156, and Week 208. IGF-1 SDS is the number of standard deviations above or below the mean IGF-1 level for a child of the same age and sex. The target range for IGF-1 SDS was 0 to +2. An IGF-1 SDS of 0 represents the population mean. If the IGF-1 SDS is < 0, then the negative score indicates an IGF-1 below the mean IGF-1 for a child of the same age and sex. If the IGF-1 SDS is > 0, then the positive score indicates an IGF-1 above the mean IGF-1 for a child of the same age and sex. A positive score of IGF-1 SDS indicates a better outcome. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical 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 |
|---|---|---|---|
| 35428884 | Result | Maniatis AK, Casella SJ, Nadgir UM, Hofman PL, Saenger P, Chertock ED, Aghajanova EM, Korpal-Szczyrska M, Vlachopapadopoulou E, Malievskiy O, Chaychenko T, Cappa M, Song W, Mao M, Mygind PH, Smith AR, Chessler SD, Komirenko AS, Beckert M, Shu AD, Thornton PS. Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results. J Clin Endocrinol Metab. 2022 Jun 16;107(7):e2680-e2689. doi: 10.1210/clinem/dgac217. |
| Label | URL |
|---|---|
| Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results. | View source |
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Patients who completed a prior phase 3 lonapegsomatropin trial were screened. Patients with poorly controlled diabetes or diabetic complications, or with evidence of closed epiphyses or known hypersensitivity to the trial medication were excluded. All inclusion/exclusion criteria were met during enrollment.
Overall, 298 subjects entered the extension trial from the parent trials: 103 subjects from the CT-301 Lonapegsomatropin group, 55 subjects from the CT-301 Genotropin group, and 140 subjects from the CT-302 Lonapegsomatropin group.
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| ID | Title | Description |
|---|---|---|
| FG000 | Lonapegsomatropin | Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH) |
| 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 | Jan 29, 2020 | Feb 20, 2024 |
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All study participants will receive TransCon hGH
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| Up to Week 208 |
| Height SDS - Change From Baseline | Change in Height Standard Deviation Score (SDS) from baseline with long-term dosing of weekly lonapegsomatropin treatment at Week 13, Week 52, Week 104, Week 156, and Week 208. Height SDS is the number of standard deviations above or below the mean height for age and sex. A Standard Deviation Score of 0 equals the population mean. If the Height SDS is < 0, then the negative score indicates a height below the mean height for a child of the same age and sex. If the Height SDS is > 0, then the positive score indicates a height above the mean height for a child of the same age and sex. A positive change from baseline in Height SDS indicates improved outcomes. | Up to Week 208 |
| Los Angeles |
| California |
| 90048 |
| United States |
| Ascendis Pharma Investigational Site | Orange | California | 92868 | United States |
| Ascendis Pharma Investigational Site | Sacramento | California | 95821 | 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 | Tallahassee | Florida | 32308 | 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 | New York | New York | 10029 | United States |
| Ascendis Pharma Investigational Site | Cleveland | Ohio | 44195 | United States |
| Ascendis Pharma Investigational Site | Oklahoma City | Oklahoma | 73104 | United States |
| Ascendis Pharma Investigational Site | Portland | Oregon | 97227 | United States |
| Ascendis Pharma Investigational Site | Portland | Oregon | 97239 | United States |
| Ascendis Pharma Investigational Site | Dallas | Texas | 75390 | United States |
| Ascendis Pharma Investigational Site | Fort Worth | Texas | 76104 | United States |
| Ascendis Pharma Investigational Site | Charlottesville | Virginia | 22908 | United States |
| Ascendis Pharma Investigational Site | Norfolk | Virginia | 23507 | United States |
| Ascendis Pharma Investigational Site | Tacoma | Washington | 98405 | United States |
| Ascendis Pharma Investigational Site | Yerevan | 0075 | Armenia |
| Ascendis Pharma Investigational Site | Clayton | Victoria | 3168 | Australia |
| Ascendis Pharma Investigational Site | Minsk | 220020 | Belarus |
| Ascendis Pharma Investigational Site | Varna | 9010 | Bulgaria |
| Ascendis Pharma Investigational Site | Tbilisi | 0159 | Georgia |
| Ascendis Pharma Investigational Site | Athens | 11527 | Greece |
| Ascendis Pharma Investigational Site | Auckland | 1010 | New Zealand |
| Ascendis Pharma Investigational Site | Gdansk | 80-952 | Poland |
| Ascendis Pharma Investigational Site | Warsaw | 02-691 | Poland |
| Ascendis Pharma Investigational Site | Izhevsk | 426009 | Russia |
| Ascendis Pharma Investigational Site | Krasnoyarsk | 660022 | Russia |
| Ascendis Pharma Investigational Site | Moscow | 117036 | Russia |
| Ascendis Pharma Investigational Site | Saint Petersburg | 194100 | Russia |
| Ascendis Pharma Investigational Site | Saratov | 410054 | 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 | Kharkiv | 61093 | Ukraine |
| Ascendis Pharma Investigational Site | Kyiv | 01021 | Ukraine |
| Ascendis Pharma Investigational Site | Odesa | 65031 | Ukraine |
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Lonapegsomatropin | Once weekly subcutaneous injection of lonapegsomatropin (TransCon hGH) |
| 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 | Race | Count of Participants | Participants |
| ||||||||||||||||||||||
| Race/Ethnicity, Customized | Ethnicity | Count of Participants | Participants |
| ||||||||||||||||||||||
| Region of Enrollment | Count of Participants | Participants |
| |||||||||||||||||||||||
| Tanner Stage | The Tanner scale is a Sexual Maturity Rating classification system used to document the development of secondary sex characteristics of children during puberty. The scale ranges from prepubertal (Stage 1) to adult (Stage 5). | Count of Participants | Participants |
| ||||||||||||||||||||||
| Height SDS | Height Standard Deviation Score (SDS) is the number of standard deviations above or below the mean height for age and sex. A Standard Deviation Score of 0 represents the population mean or average. A negative SDS indicates below average height and a positive SDS indicates above average height. | Mean | Standard Deviation | Height Standard Deviation Score |
| |||||||||||||||||||||
| IGF-1 SDS | Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS) is the number of standard deviations above or below the mean IGF-1 level for age and sex. A standard deviation score of 0 represents the population mean. | Mean | Standard Deviation | IGF-1 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 | Number of Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability] | Long-term safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment | Posted | Count of Participants | Participants | Up to Week 208 |
|
|
| |||||||||||||||||||||||||||
| Secondary | Annualized Height Velocity by Visit | Annualized height velocity (AHV) by visit with long-term dosing of weekly lonapegsomatropin treatment | A rolling baseline was used to ensure the baseline was no more than 52 weeks apart. The rolling baseline was used to calculate the annualized height velocity to ensure there was a one-year gap; from the 15-month (65 weeks) visit onwards, the baseline value used for each successive HV calculation was the patient's height at the visit 52 weeks previously. At each post-baseline visit, n is the number of subjects with non-missing baseline and a specific post-baseline. | Posted | Mean | Standard Deviation | cm/year | Up to Week 208 |
|
| ||||||||||||||||||||||||||
| Secondary | IGF-1 SDS by Visit | Insulin-like Growth Factor-1 (IGF-1) standard deviation score (SDS) by visit with long-term dosing of weekly lonapegsomatropin treatment at Week 13, Week 52, Week 104, Week 156, and Week 208. IGF-1 SDS is the number of standard deviations above or below the mean IGF-1 level for a child of the same age and sex. The target range for IGF-1 SDS was 0 to +2. An IGF-1 SDS of 0 represents the population mean. If the IGF-1 SDS is < 0, then the negative score indicates an IGF-1 below the mean IGF-1 for a child of the same age and sex. If the IGF-1 SDS is > 0, then the positive score indicates an IGF-1 above the mean IGF-1 for a child of the same age and sex. A positive score of IGF-1 SDS indicates a better outcome. | At each post-baseline visit, n is the number of subjects with non-missing baseline and specific post-baseline. | Posted | Mean | Standard Deviation | Standard Deviation Score | Up to Week 208 |
|
| ||||||||||||||||||||||||||
| Secondary | Height SDS - Change From Baseline | Change in Height Standard Deviation Score (SDS) from baseline with long-term dosing of weekly lonapegsomatropin treatment at Week 13, Week 52, Week 104, Week 156, and Week 208. Height SDS is the number of standard deviations above or below the mean height for age and sex. A Standard Deviation Score of 0 equals the population mean. If the Height SDS is < 0, then the negative score indicates a height below the mean height for a child of the same age and sex. If the Height SDS is > 0, then the positive score indicates a height above the mean height for a child of the same age and sex. A positive change from baseline in Height SDS indicates improved outcomes. | At each post-baseline visit, n is the number of subjects with non-missing baseline and specific post-baseline. | Posted | Mean | Standard Deviation | Standard Deviation Score | Up to Week 208 |
|
|
From Informed Consent Form signature up to 14 days after the final dose, up to 260 weeks.
A treatment-emergent adverse event (TEAE) was defined as any adverse event that first occurred or worsened after the first trial drug dose and before the end of the trial. Each subject was counted only once within each preferred term.
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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) | 0 | 298 | 21 | 298 | 225 | 298 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Forearm fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Lip injury | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
| |
| Diverticulitis Meckel's | Congenital, familial and genetic disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Brain stem infarction | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Epilepsy | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Generalised tonic-clonic seizure | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Serum sickness-like reaction | Immune system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cyclic vomiting syndrome | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nephrotic syndrome | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Scoliosis | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Abscess | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Gastroenteritis salmonella | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Gastrointestinal viral infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| SARS-CoV-2 test positive | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
|
The majority of enrollees were male, which is consistent with literature; Because GHD is a rare disease the study population size is modest.
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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 | Jun 3, 2019 | Apr 2, 2024 | SAP_002.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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| Adolescents (12-17 years) |
|
| Native Hawaiian or other Pacific Islander |
|
| White |
|
| Multiple |
|
| Other |
|
| Unknown |
|
| Not reported |
|
| Unknown |
|
| New Zealand |
|
| United States |
|
| Ukraine |
|
| Poland |
|
| Georgia |
|
| Australia |
|
| Belarus |
|
| Bulgaria |
|
| Russia |
|
| Canada |
|
| Turkey |
|
| Italy |
|
| Romania |
|
| Tanner Stage 3 |
|
| Tanner Stage 4 |
|
| Tanner Stage 5 |
|
|
|
|