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This is a multicenter study in Japan. Eleven sites which have already participated in the investigator-initiated clinical study (Early Access Program) will participate in this study.The objective of this study is to gain further information on the safety and efficacy of treatment with asfotase alfa.
The primary objective of this study was to collect data on the safety of repeated subcutaneous (SC) injections of asfotase alfa. There were no secondary objectives. Exploratory objectives related to the efficacy of asfotase alfa are not being reported.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Asfotase Alfa | Experimental | Patients will receive asfotase alfa by subcutaneous injection. Asfotase alfa will be administered at either 2 mg/kg 3 times per week or 1 mg/kg 6 times per week depending on investigator's discretion. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Asfotase Alfa | Biological |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs) | Adverse events are any unwanted adverse medical occurrence in patients who are treated with a medicinal drug, whether or not considered drug-related. This includes events observed in patients administered with asfotase alfa between the first dose of asfotase alfa and the completion of patient's last visit for the clinical study. | Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Kenji Fujita, MD | Medical Monitor | Study Director |
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There were 11 study sites, all in Japan; 13 patients were enrolled into this study. The first patient signed informed consent in June 2015; the study continued through November 2015 at multiple centers in Japan.
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| ID | Title | Description |
|---|---|---|
| FG000 | Overall Study | Patients enrolled in this study received a total of 6 mg/kg/week asfotase alfa by SC injection. At the Investigator's discretion, patients were continued on the dose established in the Investigator-initiated studies, receiving either 1 mg/kg asfotase alfa 6 times per week or 2 mg/kg asfotase alfa 3 times per week. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
All patients who met all of the inclusion and none of the exclusion criteria were considered the Baseline Analysis Population.
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| ID | Title | Description |
|---|---|---|
| BG000 | Overall Study | Patients enrolled in this study received a total of 6 mg/kg/week asfotase alfa by SC injection. At the Investigator's discretion, patients were continued on the dose established in the Investigator-initiated studies, receiving either 1 mg/kg asfotase alfa 6 times per week or 2 mg/kg asfotase alfa 3 times per week. |
| 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 |
| 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 Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs) | Adverse events are any unwanted adverse medical occurrence in patients who are treated with a medicinal drug, whether or not considered drug-related. This includes events observed in patients administered with asfotase alfa between the first dose of asfotase alfa and the completion of patient's last visit for the clinical study. | All patients who met all of the inclusion and none of the exclusion criteria were defined as the enrolled population, which was also the analysis population.The safety set comprised all patients who received at least 1 dose of the investigational drug. | Posted | Count of Participants | Participants | Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months. |
|
Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.
TEAEs were collected at every visit and follow-up.
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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 | Overall Study | Patients enrolled in this study received a total of 6 mg/kg/week asfotase alfa by SC injection. At the Investigator's discretion, patients were continued on the dose established in the Investigator-initiated studies, receiving either 1 mg/kg asfotase alfa 6 times per week or 2 mg/kg asfotase alfa 3 times per week. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Enterocolitis | Gastrointestinal disorders | MedDRA 17.1 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Alexion | (781) 357-9900 | clinicaltrials@alexion.com |
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| ID | Term |
|---|---|
| D007014 | Hypophosphatasia |
| D001847 | Bone Diseases |
| D012279 | Rickets |
| D010018 | Osteomalacia |
| ID | Term |
|---|---|
| D008664 | Metal Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C570710 | asfotase alfa |
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| Participants |
|
| Age, Continuous | Median | Full Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
Patients enrolled in this study received a total of 6 mg/kg/week asfotase alfa by SC injection. At the Investigator's discretion, patients were continued on the dose established in the Investigator-initiated studies, receiving either 1 mg/kg asfotase alfa 6 times per week or 2 mg/kg asfotase alfa 3 times per week.
|
|
| 0 |
| 13 |
| 9 |
| 13 |
| Injection site bruising | General disorders | MedDRA 17.1 | Systematic Assessment |
|
| Injection site erythema | General disorders | MedDRA 17.1 | Systematic Assessment |
|
| Injection site induration | General disorders | MedDRA 17.1 | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
|
| Conjunctivitis viral | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
|
| Viral rash | Infections and infestations | MedDRA 17.1 | Systematic Assessment |
|
| Feeding tube complicatino | Injury, poisoning and procedural complications | MedDRA 17.1 | Systematic Assessment |
|
| Intercostal neuralgia | Nervous system disorders | MedDRA 17.1 | Systematic Assessment |
|
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 17.1 | Systematic Assessment |
|
The Investigators are subject to certain disclosure and publication limitations. In cases of publication of the study results at conferences or on academic journals, the post-approval clinical study coordinating investigator and the study sponsor should discuss about details of publication, publication methods and presenters before decision-making
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D009140 | Musculoskeletal Diseases |
| D001851 | Bone Diseases, Metabolic |
| D002128 | Calcium Metabolism Disorders |
| D014808 | Vitamin D Deficiency |
| D001361 | Avitaminosis |
| D003677 | Deficiency Diseases |
| D044342 | Malnutrition |
| D009748 | Nutrition Disorders |