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Completed

NCT Number: NCT01920828

Gait Analysis in MPS IVA

The purpose of this study is to learn about gait (walking) characteristics in MPS IVA and possible changes in gait with enzyme replacement therapy.

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Key information

Conditions

MPS IVA Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Morquio Syndrome Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis IV Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Age range

1 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Oregon Health and Science University

Portland, Oregon, 97239, United States

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

-Diagnosis of MPS IVA

Exclusion criteria

Non-ambulatory

  • Previous enrolled in clinical trial BMN-110

Treatment and study plan

Primary outcomes

  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.

Sponsors and collaborators

Lead sponsor

Oregon Health and Science University

Other

Collaborators

  • BioMarin Pharmaceutical
  • University of Utah

Registry information

Official study title

Gait Analysis in Patients With MPS IVA Treated With Enzyme Replacement Therapy

Important dates

Study start
2013
Primary completion
2017
Study completion
2017
First posted
Aug 12, 2013
Registry last updated
Oct 17, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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