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Effect of Genestein as food supplement in patients with Sanfilippo syndrome.


- candidate number5787
- NTR NumberNTR1826
- ISRCTNISRCTN wordt niet meer aangevraagd
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR19-mei-2009
- Secondary IDs08/309 MEC AMC
- Public TitleEffect of Genestein as food supplement in patients with Sanfilippo syndrome.
- Scientific TitleEffect of Genestein as food supplement in patients with Sanfilippo syndrome (a double-blind randomised controlled trail).
- ACRONYMN/A
- hypothesisGenestein inhibits the synthesis of heparan sulphate and can possibly slow down disease progression in Sanfilippo Syndrome patients.
- Healt Condition(s) or Problem(s) studiedSanfilippo syndrome (MPS III)
- Inclusion criteria1. The patient should have a biochemically confirmed deficiency of heparan-N-sulfatase (MPSIIIA), A-N-acetylglucosaminidase (MPS IIIB) or Acetyl CoA:-glucosaminide N-acetyltransferase (MPS IIIC);
2. Patients should be able to walk soms steps independent and make some form of contact.
- Exclusion criteria1. The parent or legal representative is unwilling to participate;
2. Patient is already using Genestein;
3. Patient received an cord blood transplantation.
- mec approval receivedyes
- multicenter trialno
- randomisedyes
- masking/blindingDouble
- controlPlacebo
- groupCrossover
- Type2 or more arms, randomized
- Studytypeintervention
- planned startdate 15-jun-2009
- planned closingdate30-jul-2010
- Target number of participants30
- InterventionsThe study consists of two periods of 6 months each with a wash-out period of 1 month in between. During one of the 6 months periods patients will receive Genestein as supplement (10 mg/kg). The other 6 months period patients will receive placebo.
- Primary outcomeDoes genestein significantly decrease urinary GAGs (in particular heparan sulphate) excretion in Sanfilippo patients?
- Secondary outcome1. Does genestein decrease serum heparan sulphate concentration?
2. Does genestein decrease accumulation of heparan sulphate in skin biopsies?
3. Does hair morphology improve in MPS III patients using genestein?
4. What is the effect of genestein on behaviour?
5. Does genestein improve cognitive function?
- TimepointsBaseline, 3 month, 6 months, 7 months, 10 months and 13 months and monthly urine samples.
- Trial web siteN/A
- statusinclusion stopped: follow-up
- CONTACT FOR PUBLIC QUERIESProf.dr. F.A. Wijburg
- CONTACT for SCIENTIFIC QUERIESProf.dr. F.A. Wijburg
- Sponsor/Initiator Academic Medical Center (AMC), Amsterdam
- Funding
(Source(s) of Monetary or Material Support)
Stichting Metakids, Weeshuis der Doopsgezinden Haarlem
- PublicationsN/A
- Brief summarySanfilippo syndrome (MPS III) is an inborn errors of lysosomal degradation of the glycosaminoglycan heparan sulphate (GAGs). Accumulation of heparan sulfate, results in progressive and severe mental deterioration, which is the clinical hallmark of MPS III. Genestein (an isoflavone) is a natural component of the soy-bean. Genistein inhibits GAGs synthesis in vitro and a recent small and open-label study suggested clinical relevant effects of genestein in MPS III patients. In this study the use of genestein in MPS III patients will be compared to placebo. The study consists of two periods of 6 months each with a wash-out period of 1 month in between. During one of the 6 months periods patients will receive Genestein as supplement (10 mg/kg). The other 6 months period patients will receive placebo. During each study period patients will be studied for the effects of genestein on: urinary GAG excretion, plasma heparansulfate, tissue (skin biopsy) concentration of heparansulfate, hair structure, cognitive state and behavioural abnormalities.
- Main changes (audit trail)
- RECORD19-mei-2009 - 30-sep-2009


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