Natural History Study Update
We have now been enrolling the RCDP Natural History Study at the AIDHC in Wilmington, Delaware for over six months. It seemed in light of this achievement an update was in order. First and foremost we need to say THANK YOU to all those who have participated in the study and those who have visits scheduled to occur soon. The entire team understands that we are asking a lot of your families to travel to the site and participate in two full days of assessment. We always knew that the Rhizo community was a strong group, that understood the value of research, but your willingness to travel with your Rhizokid, sometimes across the country, to be a part of this critical study is inspiring.
When we launched this study our goal was to enroll a minimum of 15 patients, with the hope that we could get 20 patients, or more. Many questioned whether this was a feasible goal in light of the small patient population in the US, but we remained hopeful. I am excited to say that enrollment has gone amazing, the clinical team in Wilmington just finished the baseline visit on the 12th Rhizokid. In addition, four more patients are scheduled to complete their baseline assessment in the coming months, putting us at 16 patients and their families that have committed to this project!!
Overall the study itself has gone very well. The assessments that we have chosen seem to be providing a robust characterization of the different clinical manifestations of RCDP. At the end of January, Tara Smith and Vinod Kumar from MLD were on-site at the hospital. During this visit they successfully completed the first monitoring of the clinical data. This just means that they confirmed that the data was recorded and transferred into the database completely and accurately. In addition to this monitoring, the MLD and AIDHC teams had a lengthy discussion about details of the actual study to determine what was working and what could be improved.
This fall MLD asked the FDA to review the protocol for the Natural History Study and provide their feedback on the ability to use this data as a control group in a future experimental treatment trial, which has always been the ultimate goal of this study. Overall their feedback was supportive, but the Agency did have suggestions of modifications which could strengthen the data we generate. Considering the Agency’s feedback and the practical knowledge gained from assessing the first set of patients, we will be implementing some modifications to the protocol over the coming month or so. Largely this will not impact the experience of patients at their clinic visits, we just want to ensure that the data we collect is done in a way acceptable to the FDA, and that the assessments are measuring symptoms that could potentially be altered with treatment.
Management of travel was one area we always knew was going to be a challenge. We completely understood that traveling with a Rhizokid would require a travel policy that allowed for flexibility to accommodate each child and their families unique needs. That is why we contracted a travel agency, Scout, that specializes in managing patient travel. We have learned a lot from the first set of patients about what works best when arranging travel and where there are reoccurring challenges. In an attempt to improve the travel logistics portion of the study, we have put together a separate post outlining the travel policy itself and the requirements with regards to using Scout’s services for booking travel or being reimbursed. If you have, or will be, participating in the study please read the post to ensure that there are no surprises at your next visit.
Overall the last six months have been a huge success and that is all because of the support of the Rhizo families who have embraced this study. We hope the next six months and beyond will see the same level of engagement and support.