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The first clinical study in the USA with Vevesca for Type 1 Gaucher disease is to begin shortly at the New York University School of Medicine. Results of the first European multi-centre clinical study were published in the Lancet in April 2000 and the results of two further trials, one with patients taking a lower dose and one taking combination therapy with Cerezyme are due to be published shortly. Susan Lewis reports:
Oxford GlycoSciences has announced the start of its first US clinical study with Vevesca in Type 1 Gaucher disease at the New York University School of Medicine.
The study is being conducted by Dr Gregory Pastores and Dr Edwin Kolodny who run a large Gaucher Clinic there.
The trial will involve up to 14 patients with Type 1 Gaucher disease who have never received previous treatment with enzyme replacement therapy or have stopped therapy for more than three months before enrolment in the study.
They will receive one year of treatment with Vevesca which is a capsule swallowed three times a day. Patients will be monitored frequently to ensure the drug is safely and effectively managing their disease.
Dr Pastores said: 'This drug has shown promise in earlier clinical evaluations conducted in Europe and Israel. Our study is designed to provide additional information on how this new approach to managing Gaucher disease can fit in with the current treatment paradigm (regime).'
The experience gained from the US study will build on the original trial work carried out on a cohort of 28 patients in Israel, UK, Holland and Czech Republic, the results of which were published in the Lancet (a medical journal) on 29 April 2000 (described in Gaucher News, August 2000).
Apart from the original European trial, more work is due to be published on the effects of Vevesca when administered at a lower dose than employed in the first study and when given in combination with, or switching from, enzyme replacement therapy.
The former trial is with 18 Type 1 patients receiving half the original dose of Vevesca.
The other trial is with 36 patients in a combination trial where a third took Vevesca alone (switch group), a third took a combination of Vevesca and Cerezyme enzyme replacement therapy (co-administration group), and a third were on enzyme replacement alone (control group).
Initial results of the latter trial have indicated that patients were successfully maintained on Vevesca alone during a six month study period.
Of the 33 patients who completed the trial, 29 chose to enter an extension study taking Vevesca alone, comprising ten from the switch group, ten from the control group and nine from the co-administration group.
Type 3 Trial
Plans to run a trial for children with neuronopathic Gaucher disease are still underway at the National Institute of Health by Dr Roscoe Brady and Dr Raphael Schiffman.
A delay has arisen due to the fact that as children cannot easily swallow the capsule which contains the drug and the drug itself tastes very bitter, a new way of swallowing it must be found. This is currently being developed but in the the meantime it has been reported that the trial may go ahead with older children who have the capacity to swallow the capsule whole.
Registration Process in Europe and USA
Vevesca, which received orphan drug status in October 2000 from the European Commission, has now been accepted for review by the European Agency for the Evaluation of Medicinal Products. The Agency covers the European Union, Norway and Iceland.
Oxford GlycoSciences has also confirmed that it is currently working on its New Drug Application to the US Food and Drug Administration.
In both cases the company says it is not possible to predict the duration of the review process with accuracy.
Gauchers News Contents
Source: Gauchers News September 2001.
© Copyright Gauchers Association 2001