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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02456038
Recruitment Status : Completed
First Posted : May 28, 2015
Last Update Posted : March 31, 2016
Sponsor:
Collaborator:
Osaka University Graduate School of Medicine
Information provided by (Responsible Party):
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan

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

Condition or disease Intervention/treatment Phase
Hypophosphatasia Drug: Asfotase Alfa (ALXN1215) Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Hypophosphatasia (HPP)
Study Start Date : August 2014
Actual Primary Completion Date : July 2015
Actual Study Completion Date : July 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Safety

Arm Intervention/treatment
Experimental: Asfotase Alfa (ALXN1215)
Asfotase Alfa (ALXN1215) is administered 6mg/kg in total per week, divided into 3 times.
Drug: Asfotase Alfa (ALXN1215)
Other Name: ALXN1215




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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.

  2. 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

  3. 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

  4. 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

  5. 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

  6. 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



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Patients must meet one selection criteria of following "1", "2", and "3", and must meet the selection criteria of "4."

  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:
  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:

  1. Current evidence of treatable form of rickets
  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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02456038


Locations
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Japan
Kurume University Hospital
Kurume-city, Fukuoka-prefecture, Japan, 830-0011
National Hospital Organization Nagara Medical Center
Gifu-city, Gifu-prefecture, Japan, 502-8558
Hiroshima University Hospital
Hiroshima-city, Hiroshima-prefecture, Japan, 734-8551
Hokkaido University Hospital
Sapporo-city, Hokkaido-prefecture, Japan, 060-8648
Tohoku University Hospital
Sendai-city, Miyagi-prefecture, Japan, 980-8574
Niigata University Medical & Dental Hospital
Niigata-city, Niigata-prefecture, Japan, 951-8510
Osaka Medical Center and Research Institute for Maternal and Child Health
Izumi-city, Osaka-prefecture, Japan, 594-1101
Osaka University Hospital
Suita-city, Osaka-prefecture, Japan, 565-0871
Saitama Children's Medical Center
Saitama-city, Saitama-prefecture, Japan, 339-0077
Hamamatsu University Hospital
Hamamatsu-city, Shizuoka-prefecture, Japan, 431-3192
Nippon Medical School Hospital
Bunkyo-ku, Tokyo, Japan, 113-8603
Showa General Hospital
Kodaira-city, Tokyo, Japan, 187-8510
Sponsors and Collaborators
Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
Osaka University Graduate School of Medicine
Investigators
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Principal Investigator: Keiichi Ozono Osaka University Hospital
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Responsible Party: Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
ClinicalTrials.gov Identifier: NCT02456038    
Other Study ID Numbers: HPPJEAP-01
UMIN000014816 ( Other Identifier: UMIN CTR )
First Posted: May 28, 2015    Key Record Dates
Last Update Posted: March 31, 2016
Last Verified: March 2016
Additional relevant MeSH terms:
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Hypophosphatasia
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases