The aim of this study is to assess safety and efficacy of Asfotase Alfa (ALXN1215) in patients with hypophosphatasia
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
13
Kurume University Hospital
Kurume, Fukuoka, Japan
National Hospital Organization Nagara Medical Center
Gifu, Gifu, Japan
Hiroshima University Hospital
Hiroshima, Hiroshima, Japan
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
Time frame: Up to 50 months or until regulatory approval
Overall survival
Overall survival is defined as the time from birth to time of death.
Time frame: 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
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
Time frame: 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
Time frame: 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
Time frame: Up to 50 months or until regulatory approval
Profile of asfotase alfa treatment on physical growth
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Hokkaido University Hospital
Sapporo, Hokkaido-prefecture, Japan
Tohoku University Hospital
Sendai, Miyagi, Japan
Niigata University Medical & Dental Hospital
Niigata, Niigata, Japan
Osaka Medical Center and Research Institute for Maternal and Child Health
Izumi, Osaka, Japan
Osaka University Hospital
Suita, Osaka, Japan
Saitama Children's Medical Center
Saitama, Saitama, Japan
Hamamatsu University Hospital
Hamamatsu, Shizuoka, Japan
...and 2 more locations
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
Time frame: 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
Time frame: Up to 50 months or until regulatory approval