As of July, 2016, Andrew and David continue visiting Chicago every two weeks as part of the Vtesse clinical trial. Since September, 2015 they have flown back and forth for treatments as part of the second phase of the Cyclodextrin trial. This study will continue for one year and will evaluate the effectiveness and safety of Cyclodextrin in patients with Niemann-Pick Type C1 disease. Two-thirds of the patients in the study will receive the actual drug and the remaining one-third will receive sham control.
It is estimated that only about 50 people will be included in this study so we are very thankful our sons are able to be part of this important research which began in October 2015.
For more information about this trial click here or here. For an informative video about the Vtesse trial and a glimpse of what Andrew and David go through every other week in Chicago, click here. While Andrew and David are not in this video, this is exactly what they experience.