Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)

Completed

Conditions studied: Hypophosphatasia (HPP)

In brief

The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP.

Key facts

Study ID

NCT02104219

Run by

Alexion Pharmaceuticals, Inc.

People needed

32

Starts

2014-03-01

Expected to finish

2014-09-01

Last updated by the study team

2019-04-01

Who can join

Age: 5 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Documented informed consent unless patient is deceased
• Patients with Juvenile-onset HPP, defined as documented onset of first signs/symptoms at ≥ 6 months to ˂18 years
• Documented diagnosis of HPP as indicated by skeletal manifestations and low alkaline phosphatase or genotyping

You may not qualify if…

• Received treatment with asfotase alfa in the ENB-006-09 study and/or currently enrolled in the ENB-008-10 study
• Received other treatment and/or intervention to treat HPP up to 15 years old
• Other clinically significant disease

Where it is running

• Study site — Atlanta, Georgia, United States
• Study site — Indianapolis, Indiana, United States
• Study site — Boston, Massachusetts, United States
• Study site — Springfield, Missouri, United States
• Study site — St Louis, Missouri, United States
• Study site — Durham, North Carolina, United States
• Study site — Cincinnati, Ohio, United States
• Study site — Houston, Texas, United States
• Study site — Seattle, Washington, United States
• Study site — Sydney, Australia
• Study site — Winnipeg, Manitoba, Canada
• Study site — Le Kremlin-Bicêtre, France
• Study site — Paris, France
• Study site — Toulouse, France
• Study site — Rotterdam, Netherlands
• Study site — Moscow, Russia
• Study site — Istanbul, Turkey (Türkiye)
• Study site — Manchester, United Kingdom
• Study site — Sheffield, United Kingdom

Full record on ClinicalTrials.gov

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