Efficacy of genestein as food supplement in patients with the Sanfilippo syndrome (an extension study).
Completed
- Conditions
- Sanfilippo syndrome (MPS III)
- Registration Number
- NL-OMON22208
- Lead Sponsor
- Academic Medical Center Amsterdam (AMC)
- Brief Summary
/A
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 28
Inclusion Criteria
Patients participated in the initial double blind placebo controlled study on the effect of genestein in a cross-over design.
Exclusion Criteria
The parent or legal representative is unwilling to participate.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Does genestein significantly decrease urinary GAGs (in particular heparan sulphate) excretion in Sanfilippo patients?
- Secondary Outcome Measures
Name Time Method 1. Does genestein decrease serum heparan sulphate concentration?<br /><br>2. Does genestein decrease accumulation of heparan sulphate in skin biopsies?<br /><br>3. Does hair morphology improve in MPS III patients using genestein?<br /><br>4. What is the effect of genestein on behaviour?<br /><br>5. Does genestein improve neurocognitive outcome?