MPS I Clinical Trial
Official title:
Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis
Verified date | January 2018 |
Source | Children's Hospital of Eastern Ontario |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
Status | Completed |
Enrollment | 18 |
Est. completion date | December 2016 |
Est. primary completion date | March 2016 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Years to 99 Years |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
Canada | Children's Hospital of Eastern Ontario | Ottawa | Ontario |
Lead Sponsor | Collaborator |
---|---|
Children's Hospital of Eastern Ontario |
Canada,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | Ultrasound scanning technique | To determine the optimal ultrasound scanning technique for MPS patients given the potentially advanced joint contractures | one time scan | |
Secondary | Findings of synovitis or tenosynovitis | To determine whether there are ultrasound findings indicative of synovitis or tenosynovitis | one time scan | |
Secondary | Differences between MPS I and IV | To determine whether differences exist between MPS I and MPS IV | one time scan | |
Secondary | Differences between ERT and BMT | To determine whether differences exist between patients with or without Enzyme Replacement Therapy (ERT) or Bone Marrow Transplants | one time scan | |
Secondary | Differences between severe and attenuated phenotypes | To determine whether differences exist between severe and attenuated phenotypes | one time scan |
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