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This study investigates the safety, tolerability, pharmacokinetic profile (PK), and pharmacodynamic response (PD) of three different doses of ACP-001 given once-a-week compared to one dose-level of an approved daily human growth hormone product over a period of 4 weeks (4 weekly administrations versus 28 daily administrations) in adults with Growth Hormone Deficiency.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ACP-001, 0.02 mg hGH/kg/wk | Experimental | Once weekly subcutaneous injection of ACP-001 equivalent to 0.02 mg hGH/kg/week for 4 weeks |
|
| ACP-001, 0.04 mg hGH/kg/wk | Experimental | Once weekly subcutaneous injection of ACP-001 equivalent to 0.04 mg hGH/kg/week for 4 weeks |
|
| ACP-001, 0.08 mg hGH/kg/wk | Experimental | Once weekly subcutaneous injection of ACP-001 equivalent to 0.08 mg hGH/kg/week for 4 weeks |
|
| Omnitrope, 0.04 mg hGH/kg/wk | Active Comparator | Once daily subcutaneous injection of human Growth Hormone (Omnitrope) equivalent to 0.04 mg hGH/kg/week for 4 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ACP-001 (TransCon hGH) | Drug | s.c., weekly injection |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator) | Assessment of local tolerability was performed by examining injection sites by the investigator during study visits, and on the basis of records in the Patient Diary. Assessments included erythema, swelling, or pain. | Start of study treatment through Week 4 |
| Incidence of Treatment Emergent Anti-hGH Binding Antibody Formation | Number of subjects with treatment emergent anti-hGH binding antibodies | Start of study treatment through Day 42 |
| Measure | Description | Time Frame |
|---|---|---|
| Cmax of hGH | As part of the following endpoint: Pharmacokinetic (PK) profile of serum human Growth Hormone (hGH) from ACP-001 treated dose groups compared to the PK profile of hGH from the daily Omnitrope treated group. Cmax (maximum value of concentration) values at Week 4 | Days 22 to 29 |
| Emax of IGF-I |
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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 |
|---|---|---|---|---|---|---|
| Aarhus University Hospital | Aarhus | 8000 | Denmark | |||
| Charité University Hospital Berlin |
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| ID | Title | Description |
|---|---|---|
| FG000 | ACP-001, 0.02 mg hGH/kg/wk | ACP-001 (TransCon hGH): subcutaneous, weekly injection equivalent to 0.02 mg hGH/kg/wk over 4 weeks. Prior to randomization, study subjects entered a 14 to 21 day wash out period following cessation of daily growth hormone therapy. |
| FG001 | ACP-001, 0.04 mg hGH/kg/wk |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ACP-001 (TransCon hGH) |
| Drug |
s.c., weekly injection |
|
| ACP-001 (TransCon hGH) | Drug | s.c., weekly injection |
|
| Omnitrope | Drug | s.c., daily injection |
|
As part of the following endpoint: Pharmacodynamic (PD) response of serum Insulin-like Growth Factor-I (IGF-I) from ACP-001 treated dose groups compared to the PD response of IGF-I from the daily Omnitrope treated group. Emax (maximum observed response) values at Week 4 |
| Days 22 to 29 |
| Berlin |
| 12203 |
| Germany |
| University Hospital Genova | Genova | 16132 | Italy |
| Karolinska University Hospital | Stockholm | 17176 | Sweden |
ACP-001 (TransCon hGH): subcutaneous, weekly injection equivalent to 0.04 mg hGH/kg/wk over 4 weeks. Prior to randomization, study subjects entered a 14 to 21 day wash out period following cessation of daily growth hormone therapy. |
| FG002 | ACP-001, 0.08 mg hGH/kg/wk | ACP-001 (TransCon hGH): subcutaneous, weekly injection equivalent to 0.08 mg hGH/kg/wk over 4 weeks. Prior to randomization, study subjects entered a 14 to 21 day wash out period following cessation of daily growth hormone therapy. |
| FG003 | Omnitrope, 0.04 mg hGH/kg/wk | Human Growth Hormone: subcutaneous, daily injection of Omnitrope equivalent to 0.04 mg/kg/wk over 4 weeks. Prior to randomization, study subjects entered a wash out period of 14 to 21 days following cessation of daily growth hormone therapy. |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | ACP-001, 0.02 mg hGH/kg/wk | ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.02 mg hGH/kg/wk for 4 weeks |
| BG001 | ACP-001, 0.04 mg hGH/kg/wk | ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.04 mg hGH/kg/wk for 4 weeks |
| BG002 | ACP-001, 0.08 mg hGH/kg/wk | ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.08 mg hGH/kg/wk for 4 weeks |
| BG003 | Omnitrope, 0.04 mg hGH/kg/wk | Human Growth Hormone: s.c., daily injection equivalent to 0.04 mg hGH/kg/wk for 4 weeks |
| BG004 | 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 |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Weight | Mean | Standard Deviation | kg |
| |||||||||||||||
| Body Mass Index | Mean | Standard Deviation | kg/m² |
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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 Subjects Reporting Local Tolerability Events (Assessed by the Patient and Investigator) | Assessment of local tolerability was performed by examining injection sites by the investigator during study visits, and on the basis of records in the Patient Diary. Assessments included erythema, swelling, or pain. | All patients who were randomized and received at least one dose of test product were included in the Safety analysis. | Posted | Number | Number of subjects with any symptom | Start of study treatment through Week 4 |
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| Primary | Incidence of Treatment Emergent Anti-hGH Binding Antibody Formation | Number of subjects with treatment emergent anti-hGH binding antibodies | All patients who were randomized and received at least one dose of test product were included in the Safety analysis. | Posted | Number | Participants | Start of study treatment through Day 42 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Cmax of hGH | As part of the following endpoint: Pharmacokinetic (PK) profile of serum human Growth Hormone (hGH) from ACP-001 treated dose groups compared to the PK profile of hGH from the daily Omnitrope treated group. Cmax (maximum value of concentration) values at Week 4 | Pharmacokinetic and pharmacodynamic analysis was performed on all patients who had at least one measurement of the primary variable and who had attended the Day 28 study visit. Two patients in Cohort 2 (ACP-001, 0.04 mg hGH/kg/wk) were withdrawn from the study before Day 28 and were therefore excluded from the analysis. | Posted | Mean | Standard Deviation | ng/mL | Days 22 to 29 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Emax of IGF-I | As part of the following endpoint: Pharmacodynamic (PD) response of serum Insulin-like Growth Factor-I (IGF-I) from ACP-001 treated dose groups compared to the PD response of IGF-I from the daily Omnitrope treated group. Emax (maximum observed response) values at Week 4 | Pharmacokinetic and pharmacodynamic analysis was performed on all patients who had at least one measurement of the primary variable and who had attended the Day 28 study visit. Two patients in Cohort 2 (ACP-001, 0.04 mg hGH/kg/wk) were withdrawn from the study before Day 28 and were therefore excluded from the analysis. | Posted | Mean | Standard Deviation | ng/mL | Days 22 to 29 |
|
Reported Adverse Events include events starting on or after Day 0 and ending on or before Day 42.
A subject with more than one finding in a specific category was only counted once.
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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 | ACP-001, 0.02 mg hGH/kg/wk | ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.02 mg hGH/kg/wk for 4 weeks | 0 | 10 | 1 | 10 | 6 | 10 |
| EG001 | ACP-001, 0.04 mg hGH/kg/wk | ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.04 mg hGH/kg/wk for 4 weeks | 0 | 10 | 0 | 10 | 9 | 10 |
| EG002 | ACP-001, 0.08 mg hGH/kg/wk | ACP-001 (TransCon hGH): s.c., weekly injection equivalent to 0.08 mg hGH/kg/wk | 0 | 9 | 0 | 9 | 7 | 9 |
| EG003 | Omnitrope, 0.04 mg hGH/kg/wk | Human Growth Hormone: s.c., daily injection of Omnitrope equivalent to 0.04 mg hGH/kg/wk for 4 weeks | 0 | 8 | 0 | 8 | 7 | 8 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Severe Adrenal Crisis | Endocrine disorders | MedDRA 13.1 | Systematic Assessment | Patient had a history of adrenal crisis |
|
| Pleuritic Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 13.1 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fatigue | General disorders | MedDRA 13.1 | Systematic Assessment |
| |
| Application site erythema | General disorders | MedDRA 13.1 | Systematic Assessment |
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| Application site induration | General disorders | MedDRA 13.1 | Systematic Assessment |
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| Edema peripheral | General disorders | MedDRA 13.1 | Systematic Assessment |
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| Application site pruritus | General disorders | MedDRA 13.1 | Systematic Assessment |
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| Asthenia | General disorders | MedDRA 13.1 | Systematic Assessment |
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| Condition aggravated | General disorders | MedDRA 13.1 | Systematic Assessment |
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| Influenza like illness | General disorders | MedDRA 13.1 | Systematic Assessment |
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| Injection site pain | General disorders | MedDRA 13.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 13.1 | Systematic Assessment |
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| Diarrhea | Gastrointestinal disorders | MedDRA 13.1 | Systematic Assessment |
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| Abdominal distension | Gastrointestinal disorders | MedDRA 13.1 | Systematic Assessment |
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| Dyspepsia | Gastrointestinal disorders | MedDRA 13.1 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 13.1 | Systematic Assessment |
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| Toothache | Gastrointestinal disorders | MedDRA 13.1 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 13.1 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 13.1 | Systematic Assessment |
| |
| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA 13.1 | Systematic Assessment |
| |
| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | MedDRA 13.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 13.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 13.1 | Systematic Assessment |
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| Pharyngitis | Infections and infestations | MedDRA 13.1 | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA 13.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 13.1 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA 13.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 13.1 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 13.1 | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 13.1 | Systematic Assessment |
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| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 13.1 | Systematic Assessment |
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| Affect lability | Psychiatric disorders | MedDRA 13.1 | Systematic Assessment |
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| Depressed mood | Psychiatric disorders | MedDRA 13.1 | Systematic Assessment |
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| Insomnia | Psychiatric disorders | MedDRA 13.1 | Systematic Assessment |
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| Stress | Psychiatric disorders | MedDRA 13.1 | Systematic Assessment |
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| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 13.1 | Systematic Assessment |
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| Hematuria | Renal and urinary disorders | MedDRA 13.1 | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Ascendis Pharma | +45 61161658 | Asnd_registryinquiries@ascendispharma.com |
| ID | Term |
|---|---|
| D004393 | Dwarfism, Pituitary |
| ID | Term |
|---|---|
| D004392 | Dwarfism |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D001849 | Bone Diseases, Endocrine |
| D007018 | Hypopituitarism |
| D010900 | Pituitary Diseases |
| D007027 | Hypothalamic Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D004700 | Endocrine System Diseases |
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| ID | Term |
|---|---|
| D019382 | Human Growth Hormone |
| ID | Term |
|---|---|
| D013006 | Growth Hormone |
| D010908 | Pituitary Hormones, Anterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
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| Male |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Omnitrope, 0.04 mg hGH/kg/wk |
Human Growth Hormone: s.c., daily injection of Omnitrope equivalent to 0.04 mg hGH/kg/wk for 4 weeks |
|
|
Human Growth Hormone: s.c., daily injection of Omnitrope equivalent to 0.04 mg hGH/kg/wk for 4 weeks |
|
|