Idursulfase –IT clinical trials Australia

27/10/2013 20:47

My husband and I have remained in touch and have continued to work with Shire Australia, Genzyme and the Shire HCG United States to sponsor a clinical trial in Australia for MPS-II. From my 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 HCG is investigating all avenues and considering the practicality and feasibility of establishing a trial site in Australia.  I have been informed there are number of logistical issues but everyone is trying to work through the obstacles. Should Shire give the green light for a trial site in Australia there would be a patient selection process before treatment can commence.

As the selection process continues I remain cautiously optimistic and hopeful about a clinical trial to save Christian’s brain from significant cognitive involvement.  Nonetheless we considering other options for Christian to be involved in the clinical trials should Australia miss out and being selected in phase III. I am also aware that Shire has been working on starting the trials in the USA but there have been some delays.  The delays are disappointing for the many families waiting. But the safety of these trials are equally as important for family such us ours who need this hope.

 

Christian’s general health

He has been well since re-commencing intravenous ERT. During the winter and spring seasons he has had a few episodes of the usual coughs and colds. Aside from that there has not been anything too serious that requires a stay in hospital.

 

However, six weeks ago there was of course the playdoo fiasco where he had to have a general anesthetic to remove playdoo from his ears and at the same time he also had one of his grommets replaced. After several doses of antibiotics and taking very good care of his ears they are now fine. Within that same week he a bone density test and several other x-rays. They managed to get most of the tests but not the test which required him to stay perfectly still.

 

IQ test

 

The following week he had his IQ test and I am disappointed to report it did go quite as well. He was feeling unwell and just refuses to try and do any of the tasks. The psychologist didn’t want to push him too much as he was getting quite upset so we cancelled the test. She plans to visit him on the ward where he receives his infusion and have a second attempt in a few weeks. If that doesn’t work she plans to visit him at school which where I think she will have more success. He is used to the school routine and is generally more compliant.