Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia

Plain-language summary

This study evaluates and monitors over time the physical performance of adults being treated with Asfotase Alfa for hypophosphatasia (HPP). Adults aged 18 and older with pediatric-onset HPP (based on low ALP levels and/or an ALPL gene mutation) who are currently receiving Asfotase Alfa may take part. It was run as a study with no assigned phase and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Completed

Sponsor

Dr. Lothar Seefried

Start date

9/5/2018

Estimated completion

5/31/2021

Countries

Germany

Related genes

Locations

• Würzburg, Germany

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* Age ≥ 18 years at the time of enrollment
* Clinical diagnosis of pediatric-onset HPP based on low ALP (age- and sex-adjusted) and/or genetic confirmation of ALPL mutation(s), and clinical symptoms consistent with HPP
* Participant currently receiving commercial asfotase alfa treatment for HPP at the Orthopedic Institute of the Julius-Maximilians-University Würzburg, as per standard of care
* Willingness to participate in the study
* Signed informed consent

Exclusion Criteria:

* Unwillingness / anticipated inability to attend further visits
* Off-label treatment with asfotase alfa
* Current participation in an Alexion Sponsored Trial
* Experimental drug/treatment