Gait Analysis in MPS IVA

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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01920828

Enrollment

Registered

2013-08-12

Start date

2013-08-31

Completion date

2017-06-30

Last updated

2019-10-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Morquio Syndrome, MPS IVA

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.

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

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

CROSS_SECTIONAL

Eligibility

Sex/Gender

ALL

Age

1 Years to No maximum

Healthy volunteers

Inclusion criteria

-Diagnosis of MPS IVA

Exclusion criteria

Non-ambulatory * Previous enrolled in clinical trial BMN-110

Design outcomes

Primary

Measure	Time frame	Description
Measurable changes in gait	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.

Countries

United States

Outcome results