OGT 918 Drug Trial for Neuronopathic Gauchers Disease


Neuronopathic Gaucher's News Contents
Gaucher's News Contents


A clinical trial of OGT 918 in neuronopathic Gauchers disease (Types 2 and 3) is under discussion between Dr Roscoe Brady, Dr Raphael Schiffmann of the National Institutes of Health (NIH) in Washington DC, USA, and Oxford GlycoSciences, the company that produces the drug.


OGT 918, a new drug in development, is aimed at the treatment of Gauchers disease and potentially other glycosphingolipid storage disorders, e.g. Fabry's disease, late onset Tay Sachs disease and Niemann Pick Type C disease,' says Oxford GlycoSciences.

Discussions are at a preliminary stage but the proposed trial on neuronopathic Gauchers disease in children would take place at the NIH in Bethesda, just outside Washington DC. Final details of the trial have yet to be agreed.

Ingredient and Dose Changes

Before any such trial may begin, Oxford GlycoSciences must ensure that the drug is suitable for children to take. There are lots of issues to be addressed, including the ability to administer the drug as a drink - currently, OGT 918 is only available as a tablet.

Moreover, certain ingredients will have to be changed from the capsule form in order to develop the paediatric formulation.

Similarly, as is common in clinical trials involving children, the dose of the drug will have to be calculated according to the weight of each child receiving the therapy.

As with all new drugs in the development stage there are clearly defined regulatory guidelines on the conduct of such clinical trials to be followed. In the case of paediatric studies, there is a considerable amount of additional work to be undertaken.'


Neuronopathic Gaucher's News Contents
Gaucher's News Contents

Source: Gaucher's News February 2001.
© Copyright Gauchers Association 2001