An observational study in MPS IVA and Morquio Syndrome, sponsored by Oregon Health and Science University. Completed at 1 site in United States. Open to participants aged 1 Year and older. Per ClinicalTrials.gov, last updated 2019-10-17.
Sponsored by Oregon Health and Science University · Observational
The purpose of this study is to learn about gait (walking) characteristics in MPS IVA and possible changes in gait with enzyme replacement therapy.
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
63 studies on the registry are indexed under Mucopolysaccharidosis IV; 22 are open to participants now.
This study's enrollment of 10 is below the median of 117 across 32 observational studies indexed under Mucopolysaccharidosis IV.
Browse Mucopolysaccharidosis IV studies →Oregon Health and Science University is the lead sponsor of 694 studies on the registry; 133 are open to participants now.
Of its 51 completed or terminated interventional studies of FDA-regulated products, 39 (76%) have results posted.
Counted across the registry records on this site, refreshed daily.
Patients with a diagnosis of MPS IVA
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
-Diagnosis of MPS IVA
Exclusion Criteria:
Non-ambulatory
Measurable changes in gait
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.
Time frame: 36 months
The record gives a start of Aug 2013, a primary completion of Jun 2017 and a completion of Jun 2017. It does not say whether these dates are actual or estimated.
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This study is completed, as verified in Oct 2019. You cannot join it, but the record below documents what was studied.
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