|- candidate number||5787|
|- NTR Number||NTR1826|
|- ISRCTN||ISRCTN wordt niet meer aangevraagd|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||19-mei-2009|
|- Secondary IDs||08/309 MEC AMC|
|- Public Title||Effect of Genestein as food supplement in patients with Sanfilippo syndrome. |
|- Scientific Title||Effect of Genestein as food supplement in patients with Sanfilippo syndrome (a double-blind randomised controlled trail).|
|- hypothesis||Genestein inhibits the synthesis of heparan sulphate and can possibly slow down disease progression in Sanfilippo Syndrome patients.
|- Healt Condition(s) or Problem(s) studied||Sanfilippo syndrome (MPS III)|
|- Inclusion criteria||1. The patient should have a biochemically confirmed deficiency of heparan-N-sulfatase (MPSIIIA), A-N-acetylglucosaminidase (MPS IIIB) or Acetyl CoA:ƒÑ-glucosaminide N-acetyltransferase (MPS IIIC);|
2. Patients should be able to walk soms steps independent and make some form of contact.
|- Exclusion criteria||1. The parent or legal representative is unwilling to participate;|
2. Patient is already using Genestein;
3. Patient received an cord blood transplantation.
|- mec approval received||yes|
|- multicenter trial||no|
|- Type||2 or more arms, randomized|
|- planned startdate ||15-jun-2009|
|- planned closingdate||30-jul-2010|
|- Target number of participants||30|
|- Interventions||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.|
|- Primary outcome||Does genestein significantly decrease urinary GAGs (in particular heparan sulphate) excretion in Sanfilippo patients?
|- Secondary outcome||1. 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?
|- Timepoints||Baseline, 3 month, 6 months, 7 months, 10 months and 13 months and monthly urine samples.|
|- Trial web site||N/A|
|- status||inclusion stopped: follow-up|
|- CONTACT FOR PUBLIC QUERIES||Prof.dr. F.A. Wijburg|
|- CONTACT for SCIENTIFIC QUERIES||Prof.dr. F.A. Wijburg|
|- Sponsor/Initiator ||Academic Medical Center (AMC), Amsterdam|
(Source(s) of Monetary or Material Support)
|Stichting Metakids, Weeshuis der Doopsgezinden Haarlem|
|- Brief summary||Sanfilippo 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)|
|- RECORD||19-mei-2009 - 30-sep-2009|