Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP)
NCT ID: NCT02456038
Last Updated: 2016-03-31
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2
13 participants
INTERVENTIONAL
2014-08-31
2015-07-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Asfotase Alfa (ALXN1215)
Asfotase Alfa (ALXN1215) is administered 6mg/kg in total per week, divided into 3 times.
Asfotase Alfa (ALXN1215)
Interventions
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Asfotase Alfa (ALXN1215)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
1. Patient who has been already treated with Asfotae Alfa (ALXN1215) out of this clinical trial
2. Patient who has been diagnosed as HPP
3. Documented diagnosis of HPP as indicated by:
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1. Total serum alkaline phosphatase below the lower limit of normal for age
2. 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:
1. Generalized decreased ossification
2. Extreme shortening of tubular bones
3. 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:
1. History or presence of:
\- Nontraumatic post-natal fracture
\- Delayed fracture healing
2. Nephrocalcinosis or history of elevated serum calcium
3. Functional craniosynostosis
4. Respiratory compromise or rachitic chest deformity
5. Vitamin B6 dependent seizures
6. Failure to thrive
7. 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
2. Serum calcium or phosphate levels below the normal range
3. Pregnant women and nursing mothers
4. Patient who cannot enforce suitable contraceptive measures during the clinical trial
5. Prior treatment with bisphosphonates
6. Treatment with an investigational drug within 1 month prior to the start of asfotase alfa treatment
7. Current enrollment in any other study involving an investigational new drug, device or treatment for HPP (e.g., bone marrow transplantation)
8. Clinically significant disease that precludes study participation, in the opinion of the Investigator
ALL
No
Sponsors
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Osaka University Graduate School of Medicine
UNKNOWN
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
OTHER
Responsible Party
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Principal Investigators
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Keiichi Ozono
Role: PRINCIPAL_INVESTIGATOR
Osaka University Hospital
Locations
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Kurume University Hospital
Kurume, Fukuoka, Japan
National Hospital Organization Nagara Medical Center
Gifu, Gifu, Japan
Hiroshima University Hospital
Hiroshima, Hiroshima, Japan
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
Nippon Medical School Hospital
Bunkyo-ku, Tokyo, Japan
Showa General Hospital
Kodaira, Tokyo, Japan
Countries
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Other Identifiers
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UMIN000014816
Identifier Type: OTHER
Identifier Source: secondary_id
HPPJEAP-01
Identifier Type: -
Identifier Source: org_study_id