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Gait Analysis in MPS IVA

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01920828
Recruitment Status : Unknown
Verified August 2013 by Abigail Hata, Oregon Health and Science University.
Recruitment status was:  Recruiting
First Posted : August 12, 2013
Last Update Posted : August 12, 2013
University of Utah
BioMarin Pharmaceutical
Information provided by (Responsible Party):
Abigail Hata, Oregon Health and Science University

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
MPS IVA Morquio Syndrome

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

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Study Type : Observational
Estimated Enrollment : 10 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Gait Analysis in Patients With MPS IVA Treated With Enzyme Replacement Therapy
Study Start Date : August 2013
Estimated Primary Completion Date : December 2015
Estimated Study Completion Date : July 2016

Primary Outcome Measures :
  1. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with a diagnosis of MPS IVA

Inclusion Criteria:

-Diagnosis of MPS IVA

Exclusion Criteria:


  • Previous enrolled in clinical trial BMN-110

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01920828

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United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Abigail Hata, MS CGC    503-418-1061   
Principal Investigator: Abigail Hata, MS CGC         
Sponsors and Collaborators
Oregon Health and Science University
University of Utah
BioMarin Pharmaceutical

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Responsible Party: Abigail Hata, Abigail Hata, MS CGC, Oregon Health and Science University Identifier: NCT01920828     History of Changes
Other Study ID Numbers: OregonHSU-8833
First Posted: August 12, 2013    Key Record Dates
Last Update Posted: August 12, 2013
Last Verified: August 2013

Additional relevant MeSH terms:
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Mucopolysaccharidosis IV
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases