STARR Will Continue Through June 2028: Why the Additional Time Matters
If your family has been part of STARR, our natural history study in the US, you've traveled to site visits, filled out surveys, maybe gone through an EEG, or a blood draw you didn't love explaining to your kid. This, on top of everything else that comes with raising a child with STXBP1. I don't take that lightly. I want you to know what it's given us so far, and why we're asking to continue for two more years.
STARR has now seen more than 180 STXers, across multiple visits, at five sites. That's the largest longitudinal picture anyone has ever had of STXBP1. Patterns emerging from the data include: that autism and CVI show up more often in our kids than earlier estimates suggested, that active seizures show up in only a fraction of STXers at any given time, which would make counting seizures alone a poor method to tell if a novel therapy is working, and that there's no clear genetic explanation for why our kids' symptoms vary so much from one another. We are also starting to see there may be multiple patient cohorts: that STXBP1-related disorder doesn't progress the same way for every patient, which matters for how a future trial gets designed.
That's the precision we still need, and it's why we're extending STARR through June 2028. Here's what those two years make possible:
Confirming what we're seeing. More time and more data to make sure the early patterns we're finding hold up, and to know whether the tools we're using to measure progress are sensitive enough to catch a treatment effect once a trial comes. More time with each STX’er helps us distinguish real developmental change from normal ups and downs, and learn whether early abilities predict what comes next—critical for knowing when a future treatment truly changes someone's course.
Developing biomarkers. EEG, blood, sensors, remote measurement, ways of tracking change in patients that don't rely only on a clinic visit or a parent's report.
Regulatory engagement. We want STARR data to be as useful as possible in future discussions with regulators about clinical trial design, endpoints, and potentially reducing the burden on families participating on trials.
As we have been throughout the STARR Study, the Foundation will still reimburse up to $3,000 per patient per year for eligible study-related travel and expenses. We are also growing; this fall STARR adds a sixth site in Chicago at Rush University Medical Center, giving Midwest families an option closer to home. Additionally, STARR Clinic Ambassador volunteers are still available to help with giving advice as a fellow STXer parent and answering questions related to planning your trip to the clinic such as places to grab food during the day.
We're asking families who have been part of STARR to stick with the study; we're asking for the time to finish what your data has already started. For families who haven’t joined STARR, the extension of the study for two more years and/or the addition of a Midwest site will hopefully encourage you to enroll, if you are able.
And STARR's value doesn't end when the study does. These sites are becoming centers of excellence for STXBP1, with clinicians who know this condition, built to last well past 2028. And more research is coming: as new studies open at these centers, families will have the chance to take part in those too.
If you're part of STARR, thank you. Every insight we have gained has been made possible by families who choose to contribute their time, experience, and their loved one’s data to this research.
With gratitude,
Charlene Son Rigby
President, Board of Directors, STXBP1 Foundation
Learn more about the STARR Study HERE.
STARR Participant Experience Survey
The STXBP1 Foundation would like to hear from families who have participated in the STARR Natural History Study. Your feedback will help us understand what is working well, identify challenges, and improve the participant experience as the study continues. Your responses will be anonymous.