Gait Analysis in MPS IVA
The purpose of this study is to learn about gait (walking) characteristics in MPS IVA and possible changes in gait with enzyme replacement therapy.
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||Gait Analysis in Patients With MPS IVA Treated With Enzyme Replacement Therapy|
- Measurable changes in gait [ Time Frame: 36 months ] [ Designated as safety issue: No ]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.
|Study Start Date:||August 2013|
|Estimated Study Completion Date:||July 2016|
|Estimated Primary Completion Date:||December 2015 (Final data collection date for primary outcome measure)|
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
|United States, Oregon|
|Oregon Health and Science University||Recruiting|
|Portland, Oregon, United States, 97239|
|Contact: Abigail Hata, MS CGC 503-418-1061 email@example.com|
|Principal Investigator: Abigail Hata, MS CGC|