Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT02067650

Enrollment

Registered

2014-02-20

Start date

2013-09-30

Completion date

2016-12-31

Last updated

2018-01-24

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

Conditions

MPS I, MPS IV

Brief summary

Hardly any imaging studies specifically addressing the articular and periarticular structures in MPS patients are available at this point. The investigators propose to do for the first time a pilot study using musculoskeletal ultrasound in patients with various types of MPS.

Interventions

None listed

Study design

Observational model

OTHER

Time perspective

CROSS_SECTIONAL

Eligibility

Sex/Gender

ALL

Age

2 Years to 99 Years

Healthy volunteers

Inclusion criteria

* Diagnosis of Mucopolysaccharidosis * Ability to collaborate with the ultrasound assessment * Written informed consent from parents and patients over 14 years of age, assent from children 7 to 14 years

Exclusion criteria

* Inability to collaborate with the ultrasound assessment * Acute trauma to a joint that will be assessed in the 3 months preceding the study * Coexistence of other pathology that is known to affect the respective joints

Design outcomes

Primary

Measure	Time frame	Description
Power Doppler	one time exam	The primary objective is to determine whether patients with various types of MPS and at various stages of the disease exhibit abnormalities of the finger, wrist and knee joints as assessed by grey scale and Power Doppler ultrasonography.

Secondary

Measure	Time frame	Description
Ultrasound scanning technique	one time scan	To determine the optimal ultrasound scanning technique for MPS patients given the potentially advanced joint contractures
Findings of synovitis or tenosynovitis	one time scan	To determine whether there are ultrasound findings indicative of synovitis or tenosynovitis
Differences between MPS I and IV	one time scan	To determine whether differences exist between MPS I and MPS IV
Differences between ERT and BMT	one time scan	To determine whether differences exist between patients with or without Enzyme Replacement Therapy (ERT) or Bone Marrow Transplants
Differences between severe and attenuated phenotypes	one time scan	To determine whether differences exist between severe and attenuated phenotypes

Countries

Canada

Outcome results