NCT02067650

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Sep 2013

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 13, 2013

Completed
19 days until next milestone

Study Start

First participant enrolled

September 1, 2013

Completed
6 months until next milestone

First Posted

Study publicly available on registry

February 20, 2014

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2016

Completed
9 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2016

Completed
Last Updated

January 24, 2018

Status Verified

January 1, 2018

Enrollment Period

2.5 years

First QC Date

August 13, 2013

Last Update Submit

January 23, 2018

Conditions

MPS IMPS IV

Outcome Measures

Primary Outcomes (1)

  • Power Doppler

    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.

    one time exam

Secondary Outcomes (5)

  • Ultrasound scanning technique

    one time scan

  • Findings of synovitis or tenosynovitis

    one time scan

  • Differences between MPS I and IV

    one time scan

  • Differences between ERT and BMT

    one time scan

  • Differences between severe and attenuated phenotypes

    one time scan

Eligibility Criteria

Age2 Years - 99 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients with MPS I and IV

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H8L1, Canada

Location

MeSH Terms

Conditions

Mucopolysaccharidosis IV

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Johannes Roth, MD

    Children's Hospital of Eastern Ontario

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

August 13, 2013

First Posted

February 20, 2014

Study Start

September 1, 2013

Primary Completion

March 1, 2016

Study Completion

December 1, 2016

Last Updated

January 24, 2018

Record last verified: 2018-01

Locations

CA(1)