CompletedNot Applicable

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

United States32 participantsStarted 2014-03

Plain-language summary

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

Who can participate

Age range5 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 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 Exclusion Criteria: * 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

What they're measuring

Radiographic Global Impression of Change - RGI-C

Timeframe: Between Baseline (earliest available, complete, and readable x-ray set) and all available, readable post-Baseline x-ray sets during the period of patients' aged 5 to 15 years, inclusive.

Trial details

NCT IDNCT02104219

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2014-09

Results submitted2015-08-25

View on ClinicalTrials.gov More Hypophosphatasia (HPP) trials