Effect of dietary suppletion with genestein in patients with the MPS III
- Conditions
- MPS IIISanfilippo10021605
- Registration Number
- NL-OMON32555
- Lead Sponsor
- Academisch Medisch Centrum
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 30
Inclusion Criteria
The patient should have a biochemically confirmed deficiency of heparan-N-sulfatase (MPSIIIA) or A-N-acetylglucosaminidase (MPS IIIB) or Acetyl CoA:*-glucosaminide N-acetyltransferase (MPS IIIC).
Exclusion Criteria
The parent/legal representative is unwilling to participate
Patient is already using genestein
Estimated developmental age is below 1 year
Patient underwent umbrilical cord blood cell transplantation
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>-urinary GAG excretion<br /><br><br /><br><br /><br></p><br>
- Secondary Outcome Measures
Name Time Method <p>-serum heparan sulphate levels<br /><br>-GAG-accumulation in skin biopsies.<br /><br>-hair morphology<br /><br>-behaviour<br /><br>-cognitive function </p><br>