Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)

• Conditions: Hypophosphatasia

• Registration Number: NCT02496689
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

This clinical trial is being conducted in Hypophosphatasia, a bone disorder caused by gene mutation(s) resulting in bone defects. These gene mutations cause low levels of an enzyme needed to harden bone. The purpose of this study is to provide access to treatment in a disease where no approved treatment exists. This is an experimental treatment provided under specific treatment guidelines in which safety endpoints will be collected.

Detailed Description

U.S. sites participating in the expanded access program are closed to enrollment.

Study Timeline

• Study First Submitted: 2015-07-08
• Study First Submitted QC: 2015-07-10
• Study First Posted: 2015-07-14
• Status Verified: 2019-03
• Last Update Submitted: 2019-03-28
• Last Update Posted: 2019-03-29

Recruitment & Eligibility

• Status: APPROVED_FOR_MARKETING
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: EXPANDED_ACCESS
• Study Design: Not specified

Trial Locations

Locations (5)

Colorado Center for Bone Research; 🇺🇸 Lakewood, Colorado, United States

Hôpital Necker - Enfants Malades; 🇫🇷 Paris, France

Children's Hospital of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

Hôpital des Enfants; 🇫🇷 Toulouse Cedex 9, France

Hôpital Bicêtre; 🇫🇷 Le Kremlin-Bicêtre, France