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Cardiovascular disease in Sanfilippo syndrome (mucopolysaccharidosis type III)


- candidate number27567
- NTR NumberNTR6574
- ISRCTNISRCTN no longer applicable
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR18-jul-2017
- Secondary IDsW16_016 # 16.045 METC AMC
- Public TitleCardiovascular disease in Sanfilippo syndrome (mucopolysaccharidosis type III)
- Scientific TitleCardiovascular disease in Sanfilippo syndrome (mucopolysaccharidosis type III)
- ACRONYM
- hypothesisThe rationale for the proposed study is the suspicion that a significant number of MPS III patients suffer from cardiovascular disease.
- Healt Condition(s) or Problem(s) studiedCardiovascular disease, Cardiovascular disease, Sanfilippo syndrome (MPS III)
- Inclusion criteria- Enzymatic and / or biochemically confirmed MPS III A,B,C or D
- Living in the Netherlands
- Exclusion criteria- The parent or legal representative is unwilling to participate.
- Patient is unable to travel to the hospital.
- mec approval receivedyes
- multicenter trialno
- randomisedno
- group[default]
- TypeSingle arm
- Studytypeobservational
- planned startdate 3-feb-2016
- planned closingdate31-dec-2017
- Target number of participants30
- InterventionsEchocardiography
Electrocardiography
- Primary outcomeWhat is the prevalence of cardiovascular disease in (Dutch) MPS III patients?
- Secondary outcomeDo the cardiovascular abnormalities necessitate treatment?
- Timepointsnone
- Trial web siteN/A
- statusopen: patient inclusion
- CONTACT FOR PUBLIC QUERIES S.C.M. Nijmeijer
- CONTACT for SCIENTIFIC QUERIES S.C.M. Nijmeijer
- Sponsor/Initiator Academic Medical Center (AMC), Amsterdam
- Funding
(Source(s) of Monetary or Material Support)
Academic Medical Centre (AMC), Amsterdam
- Publications
- Brief summaryMucopolysaccharidose type III (MPS III) or Sanfilippo disease is a rare lysosomal storage disorder characterized by progressive neurocognitive damage with limited somatic disease. Cardiovascular involvement was believed not to be part of MPS III. However, in recent years cardiovascular disease among MPS III patients has increasingly been reported in the literature. The aim of this study is to investigate the prevalence and extent of cardiovascular disease in MPS III.
- Main changes (audit trail)
- RECORD18-jul-2017 - 20-aug-2017


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