Gait Analysis in MPS IVA

Completed

• Conditions: MPS IVA; Morquio Syndrome

• Registration Number: NCT01920828
• Lead Sponsor: Oregon Health and Science University

Brief Summary

The purpose of this study is to learn about gait (walking) characteristics in MPS IVA and possible changes in gait with enzyme replacement therapy.

Detailed Description

The purpose of this 3-year study is to conduct a longitudinal investigation using motion analysis of gait to objectively and quantitatively assess gait characteristics of MPS IVA patients. Data documenting gait characteristics and abnormalities will be gathered in patients initiating enzyme replacement therapy.

Inclusion criteria Individuals with MPS IVA diagnosed by enzyme or molecular studies Exclusion Criteria Individual with MPS IVA previously enrolled in BMN-110 Individual is non-ambulatory The individual is unable to complete longitudinal study visits at least yearly

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study Start: 2013-08
• Study First Submitted: 2013-08-08
• Study First Submitted QC: 2013-08-09
• Study First Posted: 2013-08-12
• Primary Completion: 2017-06
• Study Completion: 2017-06
• Status Verified: 2019-10
• Last Update Submitted: 2019-10-15
• Last Update Posted: 2019-10-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

-Diagnosis of MPS IVA

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Exclusion Criteria

Non-ambulatory

• Previous enrolled in clinical trial BMN-110

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Measurable changes in gait	36 months	Data acquired on patients will be compared with age-matched standardized norms. The normal data is available from a pre-existing Gait Repository, a collection of gait parameters in children without a specific diagnosis studied at Oregon Health and Science University. The results will be converted to centile charts with the aid of a biostatistician. Centile charts will be used to track statistical changes over time in the following gait parameters including but not exclusive to: velocity, cadence, step length, and base of support.

Trial Locations

Locations (1)

Oregon Health and Science University; 🇺🇸 Portland, Oregon, United States