Gait Analysis in MPS IVA

MPS IVA Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01920828
• Other study ID #: OregonHSU-8833
• Status: Completed
• Start date: August 2013
• Est. completion date: June 2017

Study information

• Verified date: October 2019
• Source: Oregon Health and Science University
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: June 2017
• Est. primary completion date: June 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year and older

Eligibility

Inclusion Criteria:

-Diagnosis of MPS IVA

Exclusion Criteria:

Non-ambulatory

• Previous enrolled in clinical trial BMN-110

Related Conditions & MeSH terms

• Morquio Syndrome
• MPS IVA
• Mucopolysaccharidosis IV

Locations

Country	Name	City	State
United States	Oregon Health and Science University	Portland	Oregon

Sponsors (3)

Lead Sponsor	Collaborator
Oregon Health and Science University	BioMarin Pharmaceutical, University of Utah

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Measurable changes in gait	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.	36 months