RCDP Therapeutic Trial Update!!

Greetings Rhizo families! We hope everyone had a great summer. It was wonderful to see some of you at the Rhizokids camp in July, and a special thanks to those who helped with the Patient-Focused Drug Development session. Our understanding is that a formal report of the day will be available through Rhizokids in the coming month.

Since July, the MLD team has been hard at work on planning the therapeutic trial for next year. This is what all of us have been working towards for nearly five years now! Currently we are focused on compiling all the data generated to date from all the safety studies and the Natural History Study into two packages that will be reviewed by the FDA. The first is for a Pre-IND meeting where we will discuss the safety data and results from the Natural History Study that will be used to select clinical endpoints in the trial. The second is the actual IND (Investigational New Drug) application that will hopfeully provide the green light for starting the trial.

Interesting fact…… we estimate that there are over 20,000 pages of data related to the mandatory PPI-1011 safety studies completed to date!!

Tara has been working hard on the protocol of the trial, which will look and feel similar to the Natural History Study but with the administration of PPI-1011. There will be some additional visits and commitments required in the trial, but we are working to make it as simple as possible. We were also recently at Nemours and met with each of the doctors involved in the different assessments to discuss preliminary data and our plans for the therapeutic trial.

Shawn has been working on the Natural History Data Analysis, and it has become clear how important having more than one timepoint is per participant. This lets us calculate a "change” (or lack thereof), in all of the assessments that are performed. This could end up being very relevant for the therapeutic trial, where effects of treatments are being made relative to a participant’s own natural history of disease progression over time. For those families who have not yet participated, it is not too late! Or, if you have one or two visits, please consider scheduling a visit over the next six months, if possible! It will greatly increase the power of the study, and make the determination of clinical benefit with PPI-1011 more robust. Click here to for information on scheduling a visit.

Lastly, stay tuned for another fireside video chat sometime in late November or early December!