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Gait Analysis in MPS IVA

Sponsor
Oregon Health and Science University (Other)
Overall Status
Completed
CT.gov ID
NCT01920828
Collaborator
University of Utah (Other), BioMarin Pharmaceutical (Industry)
10
Enrollment
1
Location
46
Duration (Months)
0.2
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase

    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

    Study Design

    Study Type:
    Observational
    Actual Enrollment :
    10 participants
    Observational Model:
    Cohort
    Time Perspective:
    Cross-Sectional
    Official Title:
    Gait Analysis in Patients With MPS IVA Treated With Enzyme Replacement Therapy
    Study Start Date :
    Aug 1, 2013
    Actual Primary Completion Date :
    Jun 1, 2017
    Actual Study Completion Date :
    Jun 1, 2017

    Outcome Measures

    Primary Outcome Measures

    1. 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.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    1 Year and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    -Diagnosis of MPS IVA

    Exclusion Criteria:

    Non-ambulatory

    • Previous enrolled in clinical trial BMN-110

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Oregon Health and Science University Portland Oregon United States 97239

    Sponsors and Collaborators

    • Oregon Health and Science University
    • University of Utah
    • BioMarin Pharmaceutical

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Oregon Health and Science University
    ClinicalTrials.gov Identifier:
    NCT01920828
    Other Study ID Numbers:
    • OregonHSU-8833
    First Posted:
    Aug 12, 2013
    Last Update Posted:
    Oct 17, 2019
    Last Verified:
    Oct 1, 2019
    Additional relevant MeSH terms:
    Osteochondrodysplasias
    Mucopolysaccharidosis IV

    Study Results

    No Results Posted as of Oct 17, 2019