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Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis

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ClinicalTrials.gov Identifier: NCT02067650
Recruitment Status : Completed
First Posted : February 20, 2014
Last Update Posted : January 24, 2018
Sponsor:
Information provided by (Responsible Party):
Johannes Roth, Children's Hospital of Eastern Ontario

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.

Condition or disease
MPS I MPS IV

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Study Type : Observational
Actual Enrollment : 18 participants
Observational Model: Other
Time Perspective: Cross-Sectional
Official Title: Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis
Actual Study Start Date : September 2013
Actual Primary Completion Date : March 2016
Actual Study Completion Date : December 2016





Primary Outcome Measures :
  1. Power Doppler [ Time Frame: 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 Outcome Measures :
  1. Ultrasound scanning technique [ Time Frame: one time scan ]
    To determine the optimal ultrasound scanning technique for MPS patients given the potentially advanced joint contractures

  2. Findings of synovitis or tenosynovitis [ Time Frame: one time scan ]
    To determine whether there are ultrasound findings indicative of synovitis or tenosynovitis

  3. Differences between MPS I and IV [ Time Frame: one time scan ]
    To determine whether differences exist between MPS I and MPS IV

  4. Differences between ERT and BMT [ Time Frame: one time scan ]
    To determine whether differences exist between patients with or without Enzyme Replacement Therapy (ERT) or Bone Marrow Transplants

  5. Differences between severe and attenuated phenotypes [ Time Frame: one time scan ]
    To determine whether differences exist between severe and attenuated phenotypes



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Ages Eligible for Study:   2 Years to 99 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with MPS I and IV
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02067650


Locations
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Canada, Ontario
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H8L1
Sponsors and Collaborators
Children's Hospital of Eastern Ontario
Investigators
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Principal Investigator: Johannes Roth, MD Children's Hospital of Eastern Ontario

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Responsible Party: Johannes Roth, Principal Investigator, Children's Hospital of Eastern Ontario
ClinicalTrials.gov Identifier: NCT02067650     History of Changes
Other Study ID Numbers: MPSUS
First Posted: February 20, 2014    Key Record Dates
Last Update Posted: January 24, 2018
Last Verified: January 2018
Additional relevant MeSH terms:
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Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases