Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

6 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-08-31

Study Completion Date

2014-09-30

Brief Summary

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The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP who served as historical controls in ENB-006-09.

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Detailed Description

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Hypophosphatasia (HPP) is a life-threatening, genetic, and ultra-rare metabolic disease characterized by defective bone mineralization and impaired phosphate and calcium regulation that can lead to progressive damage to multiple vital organs, including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. There are no approved disease-modifying treatments for patients with this disease. There is also limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.

Conditions

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Hypophosphatasia Bone Disease

Study Design

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Observational Model Type

OTHER

Study Time Perspective

RETROSPECTIVE

Study Groups

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Retrospective Case Only

Intervention Type BIOLOGICAL

Interventions

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Retrospective Case Only

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Documented informed consent/assent prior to video collection and data abstraction.
* Documented informed consent/assent in Study ALX-HPP-502.
* Patients with juvenile-onset HPP and was selected as a historical-control for Study ENB-006-09.
* Patients with at least 2 videos of basic mobility that were recorded at 2 different clinic visits when the patient was ≥5 to ≤15 ye

Minimum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No