Intrathecal clinical trial Hope is on the horizon

22/03/2014 07:11

In July 2013 we requested the pharmaceutical company Shire HGT in the U.S. to consider Australia for the Phase II/III MPS-II Intrathecal enzyme replacement trial. This clinical trial has been ongoing in the USA and the UK for a couple of years and Shire has recently began recruiting patient in Europe for the upcoming phase III trial. There is very little published data about the trial but all initial feedback has been very positive and encouraging. Unfortunately a trial site is not in Australia and I found this situation very hard to take with the reality that my son needs this medication

However, in December 2013 we were informed by Shire that they are willing to sponsor a MPS-II clinical trial in Australia. Even though this was very pleasing news we were well aware that the process still had a long way to go. The next step was to investigate which of our public hospital had the capacity to lead the trial.

I have been consulting extensively with some of our metabolic physicians to gain regular updates to the status of the trial in each hospital. It was disappointing to learn that as much as each doctor wanted to facilitate the trial, that for one reason or the other their hospital did have the resources required to proceed with the trial. 


Nonetheless, we are not one to give up especially when it comes to fighting for our son. I see the effects of hunter syndrome daily and I experienced the many challenges of this condition. I knew there was hope but it would require my husband and I to climb a massive mountain to make this trial a reality.


I kept pursuing every possible avenue to make this trial happen so that my son would not die from hunter syndrome. Also for the children that already have the condition and others not yet diagnosed. They all desperately need help and the families need hope.


In my desperation for this trial I made it my duty to consult and enquire with my son’s physician about the status of the trial on a daily basis.  We began placing significant pressure on our local hospital and as result they made investigating the trial a priority. With this persistent pressure, our local doctor has assisted in getting the Adelaide children’s hospital in South Australia to agree to support establishing a trial site. Adelaide do not currently have any MPS-II patients. However, we are very pleased that Adelaide is willing to conduct such research. 

Not only is Adelaide willing to run the trial, most importantly they have the necessary capacity and facilities required for this trial.

I am not privy to contractual arrangements between the hospital and Shire however I am very optimistic that with all the ground work that has now been done. We are well on our way to making this trial a reality.


Once an agreement is reached with the hospital and Shire this means that recruitment for the trial can begin. Eligible patients may initially need to be located in Adelaide during the initial stages of the trial. This would be for the implanting of the Intrathecal device and for clinical and neurological assessment. Monthly infusion of the ERT will also take place in Adelaide.

I will provide further updates when new information is available.