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A six month study of ACP-001, a long-acting growth hormone product, versus standard human growth hormone therapy. ACP-001 will be given once-a-week, standard human growth hormone (hGH) will be given on a daily basis. The primary aim is to demonstrate safety, pharmacokinetics and pharmacodynamics over a period of six months. A secondary objective is the comparison of height velocity (HV) of the ACP-001 treated groups to the daily hGH treatment group.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ACP-001, dose-level 1 | Experimental | Once weekly subcutaneous injection of ACP-001 |
|
| ACP-001, dose-level 2 | Experimental | Once weekly subcutaneous injection of ACP-001 |
|
| ACP-001, dose-level 3 | Experimental | Once weekly subcutaneous injection of ACP-001 |
|
| Human Growth Hormone | Active Comparator | Once daily subcutaneous injection of human Growth Hormone (rhGH) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ACP-001 | Drug | Once weekly subcutaneous injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Anti-hGH Binding Antibody Formation | Number of subjects with positive results for anti-hGH binding antibodies at two consecutive post-dose visits | Visit 2 - Visit 5 |
| Incidence of Anti-hGH Neutralizing Antibody Formation | Number of subjects with positive results for anti-hGH neutralizing antibodies at two consecutive post-dose visits | Visit 2 - Visit 5 |
| 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 anamnestic data and records in the Patient Diary. Assessments included pain, redness, bruising, swelling, and itching. Every subject was counted only once within each symptom category. | Start of study treatment through Visit 5 (Week 27) |
| Cmax of hGH | As part of the following endpoint: PK profile of serum hGH from ACP-001 treated patients compared between ACP-001 dose groups and to the pharmacokinetic (PK) profile of hGH from the daily rhGH groups during visit 1 and 3. Uncorrected Cmax (maximum value of concentration) values at Visit 3 (Week 13) | 0 hours to 168 hours at Visit 3 (Week 13) |
| AUC0-168h of hGH | As part of the following endpoint: PK profile of serum hGH from ACP-001 treated patients compared between ACP-001 dose groups and to the PK profile of hGH from the daily rhGH group during Visit 1 and Visit 3 Uncorrected AUC0-168h (area under the curve from 0h to 168h) values at Visit 3 (Week 13) | 0 hours to 168 hours at Visit 3 (Week 13) |
| E-Trough of IGF-1 |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Height Velocity | Annualized HV during treatment with ACP-001 or daily rhGH at the end of 6 months, for each ACP-001 dose group and for the daily rhGH dose group | Baseline to 6 months (Visit 5) |
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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 |
|---|---|---|---|---|---|---|
| 2nd Children City Clinic | Minsk | Belarus | ||||
| University Multiprofile Hospital for Active Treatment (UMHAT) "Sv. Marina" |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28201598 | Derived | Chatelain P, Malievskiy O, Radziuk K, Senatorova G, Abdou MO, Vlachopapadopoulou E, Skorodok Y, Peterkova V, Leff JA, Beckert M; TransCon GH Working Group. A Randomized Phase 2 Study of Long-Acting TransCon GH vs Daily GH in Childhood GH Deficiency. J Clin Endocrinol Metab. 2017 May 1;102(5):1673-1682. doi: 10.1210/jc.2016-3776. |
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| ID | Title | Description |
|---|---|---|
| FG000 | ACP-001, Dose-level 1 | Once weekly subcutaneous injection of ACP-001 ACP-001: Once weekly subcutaneous injection |
| FG001 | ACP-001, Dose-level 2 | Once weekly subcutaneous injection of ACP-001 ACP-001: Once weekly subcutaneous injection |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Human Growth Hormone | Drug | Once daily subcutaneous injection of human Growth Hormone |
|
|
As part of the following endpoint: PD profile of serum IGF-1 during Visit 1 and Visit 3 compared between the ACP-001 dose groups and to the daily rhGH group Uncorrected E-Trough (the pre-dose efficacy response) values at Week 13 |
| 0 hours to 168 hours at Visit 3 (Week 13) |
| Emax of IGF-1 | As part of the following endpoint: PD profile of serum IGF-1 during V1 and V3 compared between the ACP-001 dose groups and to the daily rhGH group | 0 hours to 168 hours at Visit 3 (Week 13) |
| AUEC0-168h of IGF-1 | As part of the following endpoint: PD profile of serum IGF-1 during V1 and V3 compared between the ACP-001 dose groups and to the rhGH group. Uncorrected AUEC0-168 (area under the efficacy curve from 0h-168h) values at Week 13 | 0 hours to 168 hours at Visit 3 (Week 13) |
| Varna |
| Bulgaria |
| Masaryk´s Hospital | Ústí nad Labem | Czechia |
| El Shatby University Hospital | Alexandria | Egypt |
| Ain Shams University Hospital | Cairo | Egypt |
| Cairo University Hospital | Cairo | Egypt |
| El Mansoura University Hospital | El Mansoura | Egypt |
| Hôpital des enfants Pellegrin | Bordeaux | France |
| Hôpital Jeanne de Flandre | Lille | France |
| Hôpital Femme-Mère-Enfant | Lyon | France |
| University Hospital Leipzig | Leipzig | Germany |
| University Children's Hospital Magdeburg | Magdeburg | Germany |
| Children's Hospital of Athens "P. A. Kyriakou" | Athens | Greece |
| Buda Children's Hospital | Budapest | Hungary |
| Heim Pal Children's Hospital | Budapest | Hungary |
| University of Pecs | Pécs | Hungary |
| University of Szeged | Szeged | Hungary |
| University Medical Hospital | Katowice | Poland |
| Medical University of Lublin | Lublin | Poland |
| Regional Hospital N°2 Rzeszow | Rzeszów | Poland |
| Children's Memorial Health Institute Warsaw | Warsaw | Poland |
| Emergency Clinical Hospital Cluj-Napoca | Cluj-Napoca | Romania |
| St. Spiridon County Clinic Emergency Hospital | Iași | Romania |
| Louis Turcanu Emergency Hospital for Children Timisoara | Timișoara | Romania |
| Federal State Budgetary Institution | Moscow | Russia |
| St. Petersburg State Pediatric Medical Academy | Saint Petersburg | Russia |
| Samara State Medical University | Samara | Russia |
| Bashkir State Medical University | Ufa | Russia |
| Children's University Hospital | Ljubljana | Slovenia |
| Ankara University School of Medicine | Ankara | Turkey (Türkiye) |
| Ege Üniversity | Izmir | Turkey (Türkiye) |
| Donetsk Regional Children Clinical Hospital | Donetsk | Ukraine |
| Kharkiv National Medical University | Kharkiv | Ukraine |
| Institute of Endocrinology and Metabolism | Kiev | Ukraine |
| Ukrainian Children Specialized Clinical Hospital | Kiev | Ukraine |
| Odessa National Medical University | Odesa | Ukraine |
| FG002 | ACP-001, Dose-level 3 | Once weekly subcutaneous injection of ACP-001 ACP-001: Once weekly subcutaneous injection |
| FG003 | Human Growth Hormone | Once daily subcutaneous injection of human Growth Hormone (rhGH) Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone |
| COMPLETED |
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| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | ACP-001, Dose-level 1 | Once weekly subcutaneous injection of ACP-001 ACP-001: Once weekly subcutaneous injection |
| BG001 | ACP-001, Dose-level 2 | Once weekly subcutaneous injection of ACP-001 ACP-001: Once weekly subcutaneous injection |
| BG002 | ACP-001, Dose-level 3 | Once weekly subcutaneous injection of ACP-001 ACP-001: Once weekly subcutaneous injection |
| BG003 | Human Growth Hormone | Once daily subcutaneous injection of human Growth Hormone (rhGH) Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone |
| 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, Categorical | Count of Participants | Participants |
| ||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||
| Baseline IGF-1 SDS (standard deviation score) | Mean | Standard Deviation | Standard Deviation Score |
| |||||||||||||||
| Baseline Height SDS | Mean | Standard Deviation | Standard Deviation Score |
| |||||||||||||||
| Screening Peak GH Values | Mean | Standard Deviation | ng/mL |
|
| 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 | Incidence of Anti-hGH Binding Antibody Formation | Number of subjects with positive results for anti-hGH binding antibodies at two consecutive post-dose visits | Safety analysis set includes all patients who receive at least one dose of planned study medication | Posted | Number | participants | Visit 2 - Visit 5 |
|
|
| |||||||||||||||||||||||||||||||||||
| Primary | Incidence of Anti-hGH Neutralizing Antibody Formation | Number of subjects with positive results for anti-hGH neutralizing antibodies at two consecutive post-dose visits | Posted | Number | participants | Visit 2 - Visit 5 |
| ||||||||||||||||||||||||||||||||||||||
| 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 anamnestic data and records in the Patient Diary. Assessments included pain, redness, bruising, swelling, and itching. Every subject was counted only once within each symptom category. | Posted | Number | Number of subjects with any symptom | Start of study treatment through Visit 5 (Week 27) |
| ||||||||||||||||||||||||||||||||||||||
| Primary | Cmax of hGH | As part of the following endpoint: PK profile of serum hGH from ACP-001 treated patients compared between ACP-001 dose groups and to the pharmacokinetic (PK) profile of hGH from the daily rhGH groups during visit 1 and 3. Uncorrected Cmax (maximum value of concentration) values at Visit 3 (Week 13) | Posted | Mean | Standard Deviation | ng/mL | 0 hours to 168 hours at Visit 3 (Week 13) |
| |||||||||||||||||||||||||||||||||||||
| Primary | AUC0-168h of hGH | As part of the following endpoint: PK profile of serum hGH from ACP-001 treated patients compared between ACP-001 dose groups and to the PK profile of hGH from the daily rhGH group during Visit 1 and Visit 3 Uncorrected AUC0-168h (area under the curve from 0h to 168h) values at Visit 3 (Week 13) | Posted | Mean | Standard Deviation | h*ng/mL | 0 hours to 168 hours at Visit 3 (Week 13) |
| |||||||||||||||||||||||||||||||||||||
| Primary | E-Trough of IGF-1 | As part of the following endpoint: PD profile of serum IGF-1 during Visit 1 and Visit 3 compared between the ACP-001 dose groups and to the daily rhGH group Uncorrected E-Trough (the pre-dose efficacy response) values at Week 13 | Posted | Mean | Standard Deviation | ng/mL | 0 hours to 168 hours at Visit 3 (Week 13) |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | Emax of IGF-1 | As part of the following endpoint: PD profile of serum IGF-1 during V1 and V3 compared between the ACP-001 dose groups and to the daily rhGH group | Posted | Mean | Standard Deviation | ng/mL | 0 hours to 168 hours at Visit 3 (Week 13) |
|
| ||||||||||||||||||||||||||||||||||||
| Primary | AUEC0-168h of IGF-1 | As part of the following endpoint: PD profile of serum IGF-1 during V1 and V3 compared between the ACP-001 dose groups and to the rhGH group. Uncorrected AUEC0-168 (area under the efficacy curve from 0h-168h) values at Week 13 | Posted | Mean | Standard Deviation | h*ng/mL | 0 hours to 168 hours at Visit 3 (Week 13) |
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Annualized Height Velocity | Annualized HV during treatment with ACP-001 or daily rhGH at the end of 6 months, for each ACP-001 dose group and for the daily rhGH dose group | Posted | Mean | Standard Deviation | cm/year | Baseline to 6 months (Visit 5) |
|
From start of the study treatment and continues until the end of the patient's participation in the study (until 4 weeks after stop of patient's study participation for serious adverse events)
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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, Dose-level 1 | Once weekly subcutaneous injection of ACP-001 ACP-001: Once weekly subcutaneous injection | 1 | 12 | 7 | 12 | ||
| EG001 | ACP-001, Dose-level 2 | Once weekly subcutaneous injection of ACP-001 ACP-001: Once weekly subcutaneous injection | 0 | 14 | 6 | 14 | ||
| EG002 | ACP-001, Dose-level 3 | Once weekly subcutaneous injection of ACP-001 ACP-001: Once weekly subcutaneous injection | 0 | 14 | 8 | 14 | ||
| EG003 | Human Growth Hormone | Once daily subcutaneous injection of human Growth Hormone (rhGH) Human Growth Hormone: Once daily subcutaneous injection of human Growth Hormone | 0 | 13 | 8 | 13 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Inguinal hernia | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Food allergy | Immune system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Hyperthermia | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Heat exhaustion | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Open wound | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Procedural dizziness | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Blood triglycerides | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Thalassemia beta | Congenital, familial and genetic disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
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| Anemia | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
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| Iron deficiency anemia | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Strabismus | Eye disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Middle ear inflammation | Ear and labyrinth disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Anal pruritis | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Diarrhea | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Hypothyroidism | Endocrine disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Secondary hypothyroidism | Endocrine disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Tonsilitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Tooth abcess | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Tracheitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA 15.1 | 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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| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
|
| More than one race |
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| Unknown or Not Reported |
|
| Romania |
|
| Hungary |
|
| Egypt |
|
| Ukraine |
|
| Belarus |
|
| Greece |
|
| Turkey |
|
| Poland |
|
| Bulgaria |
|
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