Gait Analysis in MPS IVA
Gait Analysis in Patients With MPS IVA Treated With Enzyme Replacement Therapy
1 other identifier
observational
10
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Aug 2013
Longer than P75 for all trials
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
August 1, 2013
CompletedFirst Submitted
Initial submission to the registry
August 8, 2013
CompletedFirst Posted
Study publicly available on registry
August 12, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
June 1, 2017
CompletedOctober 17, 2019
October 1, 2019
3.8 years
August 8, 2013
October 15, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
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.
36 months
Eligibility Criteria
Patients with a diagnosis of MPS IVA
You may qualify if:
- Diagnosis of MPS IVA
You may not qualify if:
- Non-ambulatory
- Previous enrolled in clinical trial BMN-110
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Oregon Health and Science Universitylead
- University of Utahcollaborator
- BioMarin Pharmaceuticalcollaborator
Study Sites (1)
Oregon Health and Science University
Portland, Oregon, 97239, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- CROSS SECTIONAL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 8, 2013
First Posted
August 12, 2013
Study Start
August 1, 2013
Primary Completion
June 1, 2017
Study Completion
June 1, 2017
Last Updated
October 17, 2019
Record last verified: 2019-10