Dr Gregory Grabowski gave an update on the trial he is carrying out giving 40mg of Fosamax a day to patients with Gauchers disease. 'I am still recruiting patients for the trial but intend to close the study in a couple of months, said Dr Grabowski at the Arlington Gaucher Conference on 2 October 1999. Dr Grabowski is Professor of Paediatrics and Molecular Medicine and Director of Human Genetics at the Children's Hospital, Cincinnati, Ohio.
'The therapy for Gaucher disease is enzyme replacement therapy', stressed Dr Grabowski. 'But the skeletal response in Gauchers patients has been at best slower than for blood and organ parameters.
'The theory is that Fosamax will bind certain crystals to the bone which will inhibit the activity and production of osteoclasts (macrophages which eat up and remove bone). We hope to find out if Fosamax will:
The 24 month trial is double blind and randomised. This way nobody knows who is getting the Fosamax and who is getting the placebo. We will also be evaluating the placebo effect.
There will be a total of 82 patients at two centres in Cincinnati and Jerusalem. They will include both male and female aged from 18 to 50 years old and the women must be pre-menopausal. All participants must have been on enzyme replacement therapy for over two years and will remain on therapy. They will be randomised by age, severity, sex and dose. 1,500mg calcium and 400iu Vitamin D a day will be given to both groups.
Bone mineral density, skeletal Xrays and chemical markers will be taken every six months and we will make an intermediate point analysis at the end of the year.'
The normal dose of Fosamax is 10mg a day compared with 40mg as given on the trial. A previous report on Dr Grabowski's trial is given in Gauchers News January 1999 . (Deutsche).
Source: Gaucher's News February 2000. © Copyright Gauchers Association 2000