Lymphocyte Gene Therapy for Mild Hunter Syndrome 
A patient will be taken "off study" if it becomes impossible to monitor treatment for any 
reason including his failure or inability to complete the necessary interval evaluations and tests. 
4.8.3 Medical Contraindication 
A patient will be taken "off study" at the time of any new medical problem, or progression 
of an existing medical problem, which makes the necessary procedures (e.g., apheresis, cell 
infusion) or tests a strict contraindication. The patient may be put back "on study" if the medical 
problem is sufficiently resolved within a time that allows for satisfactory completion of the clinical 
protocol. 
4.8.4 Iatrogenic 
A patient will be taken "off study" if there is the appearance of a hematopoietic tumor, or 
any other medical problem, that might be related to the treatment with retroviral-mediated gene 
transfer or that might be related to introduction of recombinant IDS enzyme. 
4.9 Evaluation Of Results 
At the specified intervals, and after completion of 1 year of treatment, the outcome of 
treatment will be evaluated for each patient with respect to the following results: 
4.9.1 Urine Glycosaminoglycan Excretion 
A positive response to treatment will be considered a consistent decrease in urinary 
glycosaminoglycan excretion (in comparison to elevated baseline levels obtained prior to initiation 
of lymphocyte gene therapy treatment). 
4.9.2 Organ Volumetries By CT 
A positive response to treatment will be considered a consistent decrease in liver and 
spleen size (assessed by clinical examination and also by CT-determined volumetric 
measurements) in comparison to increased volumes measured prior to initiation of lymphocyte 
gene therapy treatment. 
4.9.3 Cardiac Function 
A positive response to treatment will be considered an improvement in cardiac disease 
assessed by standard parameters of physical examination, chest x-ray, echocardiogram (valve 
leaflet thickness, regurgitation, etc.) and EKG. 
4.9.4 Respiratory Function 
A positive response to treatment will be considered a consistent improvement in 
pulmonary function test results and/or improvement in the results of an overnight sleep apnea 
study (in comparison to pretreatment baseline studies). 
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