April 17, 2009 mcfadyena

The Intrathecal Trial is Moving Ahead

The IT trial is moving ahead. I spoke with Dr. Muenzer yesterday, and he said the following information is now public info and okay to share. I wrote as quickly as I could, but I am not committing to 100% accuracy, so don’t quote me!! If you have any questions, please don’t hesitate to email me.
The trial will be accepting patients between 2-3 standard deviations below the mean ( the mean is an IQ of 100, 1 SD is 15 points), patients of IQ 55-70.
If a patient has a decline of 1-2 standard deviations, but still has IQ above 70, they can also be included.
Must be 3-8 years of age.
Dr. Escolar is no longer involved in the trial. Dr. Muenzer’s group is doing development assessments for the trial.
Infusions will be given monthly, over 6 monthes. Patient must stay at UNC for 1 week each month.
Testing for effectiveness: baseline development tests (among other tests), then 3 monthes after, and after that every 6 monthes.
Shire will be paying for child and one parent to travel to UNC for baselines, which will probably start happening in about 4-6 weeks.
The first patient will have 2 infusions before the next patient starts. 12 patients will be treated, 4 will be control. After 6 monthes, the control group can elect to start the IT enzyme. After 6 monthes of the trial, Shire will continue to pay for IT infusions of candidates.
There will be 3 different doses given: 10mg, 30mg, 100mg, starting with the lowest dose for safety.
Spread the word!!
Take care,


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