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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.
 
ClinicalTrials.gov Identifier: NCT01920828
Recruitment Status : Completed
First Posted : August 12, 2013
Last Update Posted : October 17, 2019
Sponsor:
Collaborators:
University of Utah
BioMarin Pharmaceutical
Information provided by (Responsible Party):
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
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 : August 2013
Actual Primary Completion Date : June 2017
Actual Study Completion Date : June 2017





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
Criteria

Inclusion Criteria:

-Diagnosis of MPS IVA

Exclusion Criteria:

Non-ambulatory

  • 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 ClinicalTrials.gov identifier (NCT number): NCT01920828


Locations
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United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
Sponsors and Collaborators
Oregon Health and Science University
University of Utah
BioMarin Pharmaceutical

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Responsible Party: Oregon Health and Science University
ClinicalTrials.gov Identifier: NCT01920828    
Other Study ID Numbers: OregonHSU-8833
First Posted: August 12, 2013    Key Record Dates
Last Update Posted: October 17, 2019
Last Verified: October 2019
Additional relevant MeSH terms:
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Osteochondrodysplasias
Mucopolysaccharidosis IV
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases