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

Phase 4

Completed

• Conditions: Hypophosphatasia
• Interventions: Biological: Asfotase Alfa

• Registration Number: NCT02531867
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

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.

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 Timeline

• Study Start: 2015-06
• Study First Submitted: 2015-08-11
• Study First Submitted QC: 2015-08-24
• Study First Posted: 2015-08-25
• Primary Completion: 2015-11
• Study Completion: 2015-11
• Results First Submitted: 2016-11-28
• Status Verified: 2017-03
• Results First Submitted QC: 2017-03-28
• Last Update Submitted: 2017-03-28
• Results First Posted: 2017-05-08
• Last Update Posted: 2017-05-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 13

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Asfotase Alfa	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs)	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.