Clinical Trial: Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Multicenter Study of the Safety and Efficacy of Asfotase Alfa (ALXN1215) (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Patients With Hypophospha

Brief Summary: The aim of this study is to assess safety and efficacy of Asfotase Alfa (ALXN1215) in patients with hypophosphatasia

Detailed Summary:
Sponsor: Translational Research Informatics Center, Kobe, Hyogo, Japan

Current Primary Outcome: Number of subjects with Adverse Events as an assessment of the Safety of repeated subcutaneous (SC) injections of asfotase alfa [ Time Frame: Up to 50 months or until regulatory approval ]

Safety of repeated subcutaneous (SC) injections of asfotase alfa for all treated patients


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Overall survival [ Time Frame: Up to 50 months or until regulatory approval ]
    Overall survival is defined as the time from birth to time of death.
  • 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 [ 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 for all treated patients
  • Effect of asfotase alfa treatment on ventilator-free survival: (percentage of patients who are alive and ventilator-free after receiving asfotase alfa) [ Time Frame: Up to 50 months or until regulatory approval ]
    For patients who are not mechanically ventilated at the time of enrollment, the percentage who are alive and ventilator-free after receiving asfotase alfa
  • Profile of asfotase alfa treatment on respiratory function [ Time Frame: Up to 50 months or until regulatory approval ]
    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
  • Profile of asfotase alfa treatment on physical growth [ Time Frame: Up to 50 months or until regulatory approval ]
    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
  • Effect of asfotase alfa treatment on development [ Time Frame: Up to 50 months or until regulatory approval ]
    Assessment of changes in gross motor development as measured by the developmental motor milestones for all treated patients


Original Secondary Outcome:

  • Overall survival [ Time Frame: Up to 50 months or until regulatory approval ]
    Overall survival is defined as the time from birth to time of death.
  • 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 [ 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 for all treated patients
  • Effect of asfotase alfa treatment on ventilator-free survival: (percentage of patients who are alive and ventilator-free after receiving asfotase alfa) [ Time Frame: Up to 50 months or until regulatory approval ]
    For patients who are not mechanically ventilated at the time of enrollment, the percentage who are alive and ventilator-free after receiving asfotase alfa
  • Effect of asfotase alfa treatment on respiratory function [ Time Frame: Up to 50 months or until regulatory approval ]
    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
  • Profile of asfotase alfa treatment on physical growth [ Time Frame: Up to 50 months or until regulatory approval ]
    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
  • Profile of asfotase alfa treatment on development as measured by the developmental motor milestones for all treated patients [ Time Frame: Up to 50 months or until regulatory approval ]
    Assessment of changes in gross motor development as measured by the developmental motor milestones for all treated patients


Information By: Translational Research Informatics Center, Kobe, Hyogo, Japan

Dates:
Date Received: March 26, 2015
Date Started: August 2014
Date Completion:
Last Updated: March 29, 2016
Last Verified: March 2016