Idursulfase –IT clinical trial Australia

27/10/2013 20:51

My husband and I have remained in touch and have continued to work with Shire Australia, Genzyme and the Shire HGT United States to sponsor a clinical trial in Australia for MPS-II. From our discussions setting up a Idursulfase –IT clinical site for MPS-II is a very complicated process and takes time as compare to other program such as the Sanfilippo program  (MPS-III). Nonetheless Shire HGT is investigating all avenues and considering the practicality and feasibility of establishing a trial site in Australia.  We have been informed that there are number of logistical as well as LSDP policies that are of concern to the clinical trial. 

Should we be successful in our request for a trial site in Australia there would be a patient selection process which will take approximately 12 months before treatment can commence.

As Shire USA continues the process of selecting countries for the phase III trial we remain cautiously optimistic and hopeful about a clinical trial to save Christian’s brain from significant cognitive deterioration. We are also mindful of the process and that our request was late as Shire was probably not even considering Australia before our request. I am not certain of a definite timeline for an outcome but I believe we should have an answer before the end of the year.

However, should Australia not be selected for the phase III trial we are considering other options for Christian to be involved in the a clinical trial as this is too important on so many levels! I am also aware that Shire has been working on starting the trial in the USA but there have been some delays.  The delays are disappointing for the many families waiting. But the safety of the patients are equally as important for family such us ours who need this hope.

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Idursulfase –IT clinical trial Australia

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