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OpenTrials
Completed

NCT Number: NCT02067650

Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis

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.

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Key information

Conditions

MPS I Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases MPS IV Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis IV Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Age range

2 year–99 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H8L1, Canada

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

Children's Hospital of Eastern Ontario

Other

Registry information

Important dates

Study start
2013
Primary completion
2016
Study completion
2016
First posted
Feb 20, 2014
Registry last updated
Jan 24, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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