- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02456038
Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP)
A Multicenter Study of the Safety and Efficacy of Asfotase Alfa (ALXN1215) (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in Patients With Hypophosphatasia (HPP)
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Fukuoka-prefecture
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Kurume-city, Fukuoka-prefecture, Japan, 830-0011
- Kurume University Hospital
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Gifu-prefecture
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Gifu-city, Gifu-prefecture, Japan, 502-8558
- National Hospital Organization Nagara Medical Center
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Hiroshima-prefecture
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Hiroshima-city, Hiroshima-prefecture, Japan, 734-8551
- Hiroshima University Hospital
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Hokkaido-prefecture
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Sapporo-city, Hokkaido-prefecture, Japan, 060-8648
- Hokkaido University Hospital
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Miyagi-prefecture
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Sendai-city, Miyagi-prefecture, Japan, 980-8574
- Tohoku University Hospital
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Niigata-prefecture
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Niigata-city, Niigata-prefecture, Japan, 951-8510
- Niigata University Medical & Dental Hospital
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Osaka-prefecture
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Izumi-city, Osaka-prefecture, Japan, 594-1101
- Osaka Medical Center and Research Institute for Maternal and Child Health
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Suita-city, Osaka-prefecture, Japan, 565-0871
- Osaka University Hospital
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Saitama-prefecture
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Saitama-city, Saitama-prefecture, Japan, 339-0077
- Saitama Children's Medical Center
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Shizuoka-prefecture
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Hamamatsu-city, Shizuoka-prefecture, Japan, 431-3192
- Hamamatsu University Hospital
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Tokyo
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Bunkyo-ku, Tokyo, Japan, 113-8603
- Nippon Medical School Hospital
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Kodaira-city, Tokyo, Japan, 187-8510
- Showa General Hospital
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
Patients must meet one selection criteria of following "1", "2", and "3", and must meet the selection criteria of "4."
- Patient who has been already treated with Asfotae Alfa (ALXN1215) out of this clinical trial
- Patient who has been diagnosed as HPP
- Documented diagnosis of HPP as indicated by:
- Total serum alkaline phosphatase below the lower limit of normal for age
- Ultrasonographic features of prenatal, characterized by:
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:
- Generalized decreased ossification
- Extreme shortening of tubular bones
- Hypoplastic thorax (4) Radiographic evidence of HPP, 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:
- Current evidence of treatable form of rickets
- Serum calcium or phosphate levels below the normal range
- Pregnant women and nursing mothers
- Patient who cannot enforce suitable contraceptive measures during the clinical trial
- Prior treatment with bisphosphonates
- Treatment with an investigational drug within 1 month prior to the start of asfotase alfa treatment
- Current enrollment in any other study involving an investigational new drug, device or treatment for HPP (e.g., bone marrow transplantation)
- Clinically significant disease that precludes study participation, in the opinion of the Investigator
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Asfotase Alfa (ALXN1215)
Asfotase Alfa (ALXN1215) is administered 6mg/kg in total per week, divided into 3 times.
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Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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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
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Safety of repeated subcutaneous (SC) injections of asfotase alfa for all treated patients
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Up to 50 months or until regulatory approval
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Overall survival
Time Frame: Up to 50 months or until regulatory approval
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Overall survival is defined as the time from birth to time of death.
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Up to 50 months or until regulatory approval
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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
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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
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Up to 50 months or until regulatory approval
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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
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For patients who are not mechanically ventilated at the time of enrollment, the percentage who are alive and ventilator-free after receiving asfotase alfa
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Up to 50 months or until regulatory approval
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Profile of asfotase alfa treatment on respiratory function
Time Frame: Up to 50 months or until regulatory approval
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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
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Up to 50 months or until regulatory approval
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Profile of asfotase alfa treatment on physical growth
Time Frame: Up to 50 months or until regulatory approval
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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
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Up to 50 months or until regulatory approval
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Effect of asfotase alfa treatment on development
Time Frame: Up to 50 months or until regulatory approval
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Assessment of changes in gross motor development as measured by the developmental motor milestones for all treated patients
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Up to 50 months or until regulatory approval
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Collaborators and Investigators
Collaborators
Investigators
- Principal Investigator: Keiichi Ozono, Osaka University Hospital
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- HPPJEAP-01
- UMIN000014816 (Other Identifier: UMIN CTR)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Hypophosphatasia
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AlexionEnrolling by invitationHypophosphatasia (HPP)France, Poland, United Kingdom, Germany, Spain, Saudi Arabia, United States, Canada, Russian Federation, Australia, Italy
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Hvidovre University HospitalOdense University HospitalActive, not recruitingHypophosphatasia (HPP)Denmark
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Taiwan, United Kingdom, Australia, Canada, Germany, Spain
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, France, United Kingdom, Canada, Netherlands, Russian Federation, Turkey, Australia
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada, United Arab Emirates, United Kingdom
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Alexion Pharmaceuticals, Inc.RecruitingHypophosphatasiaUnited States, Japan, Italy, Turkey, Australia, Germany, United Kingdom, Argentina, France, Canada, India
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Alexion Pharmaceuticals, Inc.Not yet recruiting
Clinical Trials on Asfotase Alfa (ALXN1215)
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Alexion PharmaceuticalsWithdrawn
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Alexion Pharmaceuticals, Inc.RecruitingHypophosphatasiaUnited States, Japan, Italy, Turkey, Australia, Germany, United Kingdom, Argentina, France, Canada, India
-
AlexionRecruiting
-
Alexion PharmaceuticalsCompletedHypophosphatasiaUnited States, Germany
-
Alexion Pharmaceuticals, Inc.Xcenda, LLCActive, not recruitingHypophosphatasiaUnited States
-
Alexion PharmaceuticalsCompleted
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Alexion PharmaceuticalsApproved for marketingHypophosphatasiaUnited States, France
-
Alexion PharmaceuticalsCompletedHypophosphatasiaUnited States, United Arab Emirates, United Kingdom
-
Alexion PharmaceuticalsCompletedHypophosphatasiaUnited States, Spain, Australia, United Kingdom, Italy, France, Canada, Germany, Japan, Russian Federation, Saudi Arabia, Turkey
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada, United Arab Emirates, United Kingdom