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

Hypophosphatasia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02531867
• Other study ID #: AA-HPP-409
• Status: Completed
• Phase: Phase 4
• First received: August 11, 2015
• Last updated: August 4, 2016
• Start date: July 2015
• Est. completion date: November 2015

Study information

• Verified date: August 2016
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: Japan: Pharmaceuticals and Medical Devices Agency
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 13
• Est. completion date: November 2015
• Est. primary completion date: November 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hypophosphatasia

Intervention

Biological:
• Asfotase Alfa

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The safety of asfotase alfa will be assessed by routine monitoring of patients about Adverse Events (AEs) including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs)	The severity (mild, moderate or severe)of each AE will be assessed by Common Terminology Criteria for Adverse Events (CTCAE) v. 4.03.	Up to 3 months	No