Trial for new Enzyme Replacement Therapy for Gaucher Disease to take place in Israel


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A clinical trial started in July 2004 for a new enzyme replacement therapy for Gaucher Disease developed by the pharmaceutical company, Transkaryotic Therapies Inc (TKT).


Transkaryotic Therapies Inc announced on 26 July 2004 that it has completed enrolment of an open-label Phase I/II study to evaluate the safety and clinical activity of GA-GCB, its investigational enzyme replacement therapy for the treatment of Gaucher disease.

The study has enrolled twelve patients with Type 1 Gaucher disease from several countries. The company expects to release data from the trial in the second half of 2005.

The lead investigator of the study, Prof Ari Zimran of the Shaare Zedek Medical Center in Jerusalem, Israel, stated: 'I am excited to lead the first Gaucher study evaluating an alternative enzyme replacement therapy for Gaucher patients. 'Since this therapeutic protein is produced in human cells, I believe it may have important effects in treating the disease.

'The primary objective of this clinical trial is to evaluate the safety of the drug when administered at a dose of 60 units per kilogram of body weight every two weeks for a total of 40 weeks (20 intravenous infusions) to 12 patients with Gaucher disease.

'The secondary objective is to evaluate the clinical activity in these patients, as measured by improvement in hematological parameters and reductions in liver and spleen volume. It is an open-label Phase I/II study.'

Transkaryotic Therapies Inc (also known as TKT) is a bio-pharmaceutical company primarily focused on researching, developing and commercializing treatments for rare diseases caused by protein deficiencies. The company also markets Replagal, an enzyme replacement therapy for Fabry disease and is also developing a treatment for Hunter syndrome.

The company was founded in 1988 and is based in Cambridge, Massachusetts, USA


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Source: Gauchers News October 2004.
© Copyright Gauchers Association 2004.