Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| UMIN000014816 | Other Identifier | UMIN CTR |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Osaka University Graduate School of Medicine | UNKNOWN |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The aim of this study is to assess safety and efficacy of Asfotase Alfa (ALXN1215) in patients with hypophosphatasia
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Asfotase Alfa (ALXN1215) | Experimental | Asfotase Alfa (ALXN1215) is administered 6mg/kg in total per week, divided into 3 times. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Asfotase Alfa (ALXN1215) | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of subjects with Adverse Events as an assessment of the Safety of repeated subcutaneous (SC) injections of asfotase alfa | Safety of repeated subcutaneous (SC) injections of asfotase alfa for all treated patients | Up to 50 months or until regulatory approval |
| Measure | Description | Time Frame |
|---|---|---|
| Overall survival | Overall survival is defined as the time from birth to time of death. | Up to 50 months or until regulatory approval |
| Effect of asfotase alfa treatment on skeletal manifestations of HPP as measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale |
Not provided
Inclusion Criteria:
Patients must meet one selection criteria of following "1", "2", and "3", and must meet the selection criteria of "4."
1) severe short extremities (femur length <-4SD in second and third trimesters) 2) extending into the metaphysis (femur metaphysis length or femur length >0.33) 3) craniotabes 4) Hypoplastic thorax (Thoracic or abdominal circumference <0.6) (3) Computed tomographic findings of prenatal, characterized by:
1) Flared and frayed metaphyses 2) Severe, generalized osteopenia 3) Widened growth plates 4) Areas of radiolucency or sclerosis (5) Two or more of the following HPP-related findings:
History or presence of:
- Nontraumatic post-natal fracture
- Delayed fracture healing
Nephrocalcinosis or history of elevated serum calcium
Functional craniosynostosis
Respiratory compromise or rachitic chest deformity
Vitamin B6 dependent seizures
Failure to thrive
Premature tooth loss (6) Patient who have the mutation of tissue non-specific ALP gene 4. Parent or legal guardian(s) must provide written informed consent prior to any study procedures being performed and must be willing to comply with all study-required procedures
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Keiichi Ozono | Osaka University Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kurume University Hospital | Kurume | Fukuoka | 830-0011 | Japan | ||
| National Hospital Organization Nagara Medical Center |
Not provided
| ID | Term |
|---|---|
| D007014 | Hypophosphatasia |
| ID | Term |
|---|---|
| D008664 | Metal Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| C570710 | asfotase alfa |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Effect of asfotase alfa treatment on skeletal manifestations of HPP as measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale for all treated patients |
| Up to 50 months or until regulatory approval |
| Effect of asfotase alfa treatment on ventilator-free survival: (percentage of patients who are alive and ventilator-free after receiving asfotase alfa) | For patients who are not mechanically ventilated at the time of enrollment, the percentage who are alive and ventilator-free after receiving asfotase alfa | Up to 50 months or until regulatory approval |
| Profile of asfotase alfa treatment on respiratory function | Effect of asfotase alfa treatment on respiratory function as measured by ventilator status, time on respiratory support (including time on ventilator or supplemental oxygen), ventilator rate or oxygen volume, ventilator pressures, and fraction of inspired oxygen (FiO2) for all treated patients | Up to 50 months or until regulatory approval |
| Profile of asfotase alfa treatment on physical growth | Effect of asfotase alfa treatment on physical growth as measured by body weight, length, arm span, head circumference, and chest circumference for all treated patients | Up to 50 months or until regulatory approval |
| Effect of asfotase alfa treatment on development | Assessment of changes in gross motor development as measured by the developmental motor milestones for all treated patients | Up to 50 months or until regulatory approval |
| Gifu |
| Gifu |
| 502-8558 |
| Japan |
| Hiroshima University Hospital | Hiroshima | Hiroshima | 734-8551 | Japan |
| Hokkaido University Hospital | Sapporo | Hokkaido-prefecture | 060-8648 | Japan |
| Tohoku University Hospital | Sendai | Miyagi | 980-8574 | Japan |
| Niigata University Medical & Dental Hospital | Niigata | Niigata | 951-8510 | Japan |
| Osaka Medical Center and Research Institute for Maternal and Child Health | Izumi | Osaka | 594-1101 | Japan |
| Osaka University Hospital | Suita | Osaka | 565-0871 | Japan |
| Saitama Children's Medical Center | Saitama | Saitama | 339-0077 | Japan |
| Hamamatsu University Hospital | Hamamatsu | Shizuoka | 431-3192 | Japan |
| Nippon Medical School Hospital | Bunkyo-ku | Tokyo | 113-8603 | Japan |
| Showa General Hospital | Kodaira | Tokyo | 187-8510 | Japan |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |