Zavesca Trial for Type 3 Gauchers Disease Starts in the UK

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This July saw the start of a screening process of children with Type 3 Gauchers disease at Great Ormond Street Hospital to decide if they were suitable to enter the Zavesca (OGT 918) trial, writes Tanya Collin-Histed.

Each child with Type 3 Gauchers disease participating in the Zavesca trial has to undertake a number of baseline (initial) checks at Great Ormond Street Hospital which include blood tests, height, weight, tremor assessment, abdominal MRI, quality of life questionnaire, neurological assess-ment and neuropsychological tests.

The initial baseline screening is carried out over a period of three days. The children then travel to Plymouth to undergo eye movement and hearing measurements with Prof Chris Harris and Pauline Campbell at the Institute of Neuroscience at the University of Plymouth.

Following successful completion of the tests each child is randomly chosen through a special procedure to either take Zavesca or enter in the control study when they do not take the drug. All children will continue taking enzyme replacement therapy. Two out of three children are picked to take Zavesca. They then visit Great Ormond Street Hospital to collect the first three months supply of the drug.

The first child was randomised at the end of July with the majority of the other children being chosen through August and September. Those children not taking Zavesca will continue to visit Great Ormond Street Hospital and Plymouth Hospital on a three to six monthly basis for regular assessments. These children will provide a control group for the clinical trial in order to find out if the drug is helpful by comparing the data between the two groups of children.

The parents of children taking Zavesca are required to keep a daily nutritional diary and record when the capsule is swallowed which should be three times a day. The trial nurse co-ordinator Elin Davies-Pope will maintain regular contact with the parents of the children taking Zavesca by phoning weekly between hospital visits. The trial will run for one year with a further one year extension. All children completing the main study (those taking Zavesca and those not taking it) will be eligible to enter the exten-sion study and all will receive the drug if it proves to be successful .

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Source: Gauchers News October 2003.
© Copyright Gauchers Association Ltd 2003.