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

NCT ID: NCT02531867

Last Updated: 2017-05-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 13 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-06-30
• Study Completion Date: 2015-11-30

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.

Conditions

Hypophosphatasia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Asfotase Alfa

Intervention Type: BIOLOGICAL

Interventions

Asfotase Alfa

Intervention Type: BIOLOGICAL

Other Intervention Names

Strensiq

Eligibility 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.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Kenji Fujita, MD

Role: STUDY_DIRECTOR

Medical Monitor

Other Identifiers

AA-HPP-409

Identifier Type: -

Identifier Source: org_study_id