Clinical Trial: Gait Analysis in MPS IVA

Study Status: Active, not recruiting
Recruit Status: Unknown status
Study Type: Observational

Official Title: Gait Analysis in Patients With MPS IVA Treated With Enzyme Replacement Therapy

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 Summary:

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


Sponsor: Oregon Health and Science University

Current Primary Outcome: Measurable changes in gait [ Time Frame: 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.


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Oregon Health and Science University

Dates:
Date Received: August 8, 2013
Date Started: August 2013
Date Completion: July 2016
Last Updated: August 9, 2013
Last Verified: August 2013