Gait Analysis in MPS IVA

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2013 by Oregon Health and Science University
University of Utah
BioMarin Pharmaceutical
Information provided by (Responsible Party):
Abigail Hata, Oregon Health and Science University Identifier:
First received: August 8, 2013
Last updated: August 9, 2013
Last verified: August 2013
The purpose of this study is to learn about gait (walking) characteristics in MPS IVA and possible changes in gait with enzyme replacement therapy.

Morquio Syndrome

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Gait Analysis in Patients With MPS IVA Treated With Enzyme Replacement Therapy

Resource links provided by NLM:

Further study details as provided by Oregon Health and Science University:

Primary Outcome Measures:
  • Measurable changes in gait [ Time Frame: 36 months ] [ Designated as safety issue: No ]
    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.

Estimated Enrollment: 10
Study Start Date: August 2013
Estimated Study Completion Date: July 2016
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
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


Ages Eligible for Study:   1 Year and older
Genders Eligible for Study:   Both
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01920828

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
  More Information

Responsible Party: Abigail Hata, Abigail Hata, MS CGC, Oregon Health and Science University Identifier: NCT01920828     History of Changes
Other Study ID Numbers: OregonHSU-8833 
Study First Received: August 8, 2013
Last Updated: August 9, 2013
Health Authority: United States: Data and Safety Monitoring Board

Additional relevant MeSH terms:
Mucopolysaccharidosis IV
Bone Diseases
Bone Diseases, Developmental
Carbohydrate Metabolism, Inborn Errors
Connective Tissue Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Musculoskeletal Diseases processed this record on May 26, 2016