Clinical Trial: 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 Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: A Single-Center, Non-interventional Substudy of ALX-HPP-502 to Assess Functional Natural History Data of Patients With Juvenile Historical Controls in ENB-006-09

Brief Summary: 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.

Detailed Summary: 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.
Sponsor: Alexion Pharma GmbH

Current Primary Outcome: Modified Performance-Oriented Mobility Assessment-Gait Subtest (MPOMA-G) - Change From Baseline to Last Overall [ Time Frame: The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive. ]

The MPOMA-G is a 5-component assessment that is used to evaluate gait performance. The first component has 4 sub-components. For 2 components and 2 sub-components, scores of 0 or 1 are assigned while scores of 0, 1 or 2 are assigned to the rest of the 2 components and 2 sub-components based on type of ambulation pattern observed. The maximum total score of 12 points = no impairment and 0 points = worst impairment.


Original Primary Outcome: Characterize gait performance over time in untreated historical-control patients [ Time Frame: 10 years ]

Metric to be used is the modified Tinetti Performance Oriented Mobility Assessment, gait subtest (POMA-G) score


Current Secondary Outcome: Performance-Oriented Mobility Assessment-Gait Subtest (POMA-G) - Change From Baseline to Last Overall [ Time Frame: The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive. ]

The POMA-G is a 7-component assessment that is used to evaluate gait performance. The second component has 4 sub-components. Scores of 0, 1 or 2 are assigned to 2 components while scores of 0 or 1 are assigned to the rest of the 4 components and 4 sub-components based on type of ambulation pattern observed. The maximum total score of 12 points = no impairment and 0 points = worst impairment.


Original Secondary Outcome:

Information By: Alexion Pharma GmbH

Dates:
Date Received: September 5, 2014
Date Started: August 2014
Date Completion:
Last Updated: October 22, 2015
Last Verified: October 2015