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Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan

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: NCT02531867
Recruitment Status : Completed
First Posted : August 25, 2015
Results First Posted : May 8, 2017
Last Update Posted : May 8, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
This is a multicenter study in Japan. Eleven sites which have already participated in the investigator-initiated clinical study (Early Access Program) will participate in this study.The objective of this study is to gain further information on the safety and efficacy of treatment with asfotase alfa.

Condition or disease Intervention/treatment Phase
Hypophosphatasia Biological: Asfotase Alfa Phase 4

Detailed Description:
The primary objective of this study was to collect data on the safety of repeated subcutaneous (SC) injections of asfotase alfa. There were no secondary objectives. Exploratory objectives related to the efficacy of asfotase alfa are not being reported.

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Post-Approval Clinical Study for Asfotase Alfa (Human Recombinant Tissue-nonspecific Alkaline Phosphatase Fusion Protein) Treatment for Patients With Hypophosphatasia (HPP) in Japan
Actual Study Start Date : June 2015
Primary Completion Date : November 2015
Study Completion Date : November 2015

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: Asfotase Alfa
Patients will receive asfotase alfa by subcutaneous injection. Asfotase alfa will be administered at either 2 mg/kg 3 times per week or 1 mg/kg 6 times per week depending on investigator's discretion.
Biological: Asfotase Alfa
Other Name: Strensiq


Outcome Measures

Primary Outcome Measures :
  1. Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs) [ Time Frame: Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months. ]
    Adverse events are any unwanted adverse medical occurrence in patients who are treated with a medicinal drug, whether or not considered drug-related. This includes events observed in patients administered with asfotase alfa between the first dose of asfotase alfa and the completion of patient's last visit for the clinical study.


Eligibility Criteria

Information from the National Library of Medicine

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

Inclusion Criteria:

  1. Patient or parent (or legal guardian) must provide written informed consent prior to the performance of any study-related procedures and must be willing to comply with study procedures. Where appropriate and required by local regulations, patient assent for participation must also be obtained.
  2. Patient has completed the investigator-initiated clinical study (HPPJEAP-01) protocol for asfotase alfa

Exclusion Criteria:

  1. Patient has a documented form of rickets caused by a condition other than HPP, including, but not limited to, rickets caused by 25(OH) vitamin D deficiency
  2. Patient has serum calcium and/or phosphorus levels below the normal range
  3. Patient is pregnant or lactating
  4. Patient received treatment with bisphosphonates within 2 years prior to the Screening visit
  5. Patient has a documented sensitivity to any of the components of asfotase alfa
  6. Patient is currently enrolled in any other program or clinical study involving an investigational new drug, device, or treatment for HPP (eg, bone marrow transplantation)
  7. Patient has clinically significant other disease in the opinion of the Investigator, defined as any other non HPP-related condition for which the patient is considered medically unstable.
Contacts and Locations

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): NCT02531867


Sponsors and Collaborators
Alexion Pharmaceuticals
Investigators
Study Director: Kenji Fujita, MD Medical Monitor
More Information

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02531867     History of Changes
Other Study ID Numbers: AA-HPP-409
First Posted: August 25, 2015    Key Record Dates
Results First Posted: May 8, 2017
Last Update Posted: May 8, 2017
Last Verified: March 2017

Keywords provided by Alexion Pharmaceuticals:
HPP
Bone Disease
Soft Bones
Low Alkaline Phosphatase
Genetic Metabolic Disorder
Alkaline Phosphatase
Rickets
Osteomalacia

Additional relevant MeSH terms:
Hypophosphatasia
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Immunoglobulin G
Immunologic Factors
Physiological Effects of Drugs