A little boy who is always smiling (getting ready for IT clinical trial)

14/08/2014 21:05

Christian has been sick for nearly two weeks. He has had bronchiolitis, an ear infection and the flu.  We moved house and while we were unpacking he accidentally stepped on a steel plate and cut his foot badly. This was very difficult for him and us because he didn’t want to keep his dressing on his foot and we had to keep insisting he keeps it on. Only within a few days of feeling better he went back to school and his teacher reported he did very well but the next day he had to go into surgery to install new grommets and to do a hearing test called an Auditory Brainstem Response (ABR). The doctors need to know the level of his hearing loss so he can be fitted with the right device and therefore ready to participate in the upcoming Phase II/III Idulrsulfase IT Trial.

Whilst we have pioneered this clinical trial to Australia and have been working consistently to see that it proceeds, he still needs to meet the eligibility criteria for the trial. One of the criteria is that each participant has sufficient hearing. Therefore we are currently investigating hearing devices called the Baha Implant and the Bone bridge implant.

To determine if Christian qualifies for either of these devices requires several hearing tests be done under general anesthetic including a CT scan and two ABRs. But as with any medical procedure there are always risks particularly when your child which suffers from mucopolysaccharidosis is placed under general anesthetic. This is mainly because of the airway and respiratory concerns but with professional care they can be overcome. Christian has many general anesthetics and so far he has been fine but we still get concern.

The initial feedback from the audiologist is that this latest test went well and with some detailed assessment will enable the correct selection of the hearing device our Christian needs to have a normal life and have a chance to participate in a life changing trial in the coming weeks.