MLD on location in Wilmington!
A big hello to the RCDP community, live from the Alfred I duPont Hospital for Children in Wilmington, Delaware! Also, a big welcome to new participants who have recently joined the study. We cannot emphasize enough how critical it is that participation in the Natural History Study continues, because the information we are gathering will be used to inform on possible endpoints and act as a control for the drug intervention trial. We know that Covid has caused hesitancy to travel, resulting in delays in visits, but hopefully we are on the tail end of the pandemic and can resume normal activities once again.
We (Tara Smith, Kaeli Knudsen, and Shawn Ritchie) have been here this past week monitoring the Natural History clinical data. This is a regulatory requirement that the data collected during your visits and recorded in the source documents be verified in the electronic database, and that the surveys have been completed. We are also working with Dr. Bober and a biostatistician to start analyzing some of the data, with the goal of publishing the findings and selecting appropriate clinical endpoints for the intervention trial. It is amazing to see the massive amount of data that is available, and it speaks to the commitment of not only participants travelling to the site, but of Dr. Bober’s team as well.
Longitudinal visits (more than one visit per subject) are emerging as an essential part of the Natural History Study, because the ability to measure multiple datapoints in the same participant over time will likely be required to detect potential changes once on treatment. An extra thanks to those participants who have completed multiple visits! For those with only the baseline completed, we strongly encourage you to complete the full study and contact the hospital to book your remaining visit(s) if possible. We are committed to making the travel process as easy as possible within the travel policy, and hope that since bringing travel logistics under the roof at MLD, travel experiences have improved.
The preclinical safety studies on the drug are also all complete and we are just awaiting the final reports. A large multi-kilogram batch of drug is currently being synthesized, with the Phase I adult human clinical trial starting to take shape. We will provide another update soon once the safety reports are in and we have the Phase I study finalized.
Thank-you for your continued support and patience. Without your participation, it would be impossible for us to continue our development of this therapy at MLD.
Sincerely,
The MLD team.