Clinical Trials and Sharing Personal Experiences
James Goss PhD, Scientific Director & Charlene Son Rigby, Co-Founder and President
This is a momentous time for the STXBP1 community. The first clinical trial for a genetic therapy for STXBP1 disorders has started. And others are in the works.
A few courageous families will be the first participants in the Capsida trial. Let's support them as best we can, including allowing them privacy during this time.
Families and the community may have questions about what is appropriate to share. Here we provide considerations on what to share, based on learnings from other rare diseases.
Many caregivers and families use social media to connect with others who have similar experiences. It helps people feel supported and not alone. Our community has social media groups on Facebook, WhatsApp, and other platforms where members share information, experiences, and thoughts. It makes sense that people might talk about clinical trials in these conversations. Sharing information about your experience is always your choice and please make this choice thoughtfully.
Clinical trials are very important for creating new treatments for STXBP1: trials teach us whether a given treatment is safe and works well. To do this, trials are carefully planned to collect reliable information about a new treatment's benefits and side effects. This means the information gathered during the trial needs to stay as unbiased as possible.
Here are some things to think about when sharing your trial experience on social media based on what has been seen in other conditions, as sharing can influence a trial’s results:
What you post online might change how other people notice or talk about their own child’s symptoms. This can make it harder to tell if the treatment is actually having a positive effect.
When people talk about side effects online and get medical advice there instead of telling the study team, clinicians may worry about safety.
If too much information gets shared publicly, it could hurt the study's reliability. This could hurt the trial’s objectivity and could have a negative impact on the clinical findings. It might also mean the trial might have to be repeated.
Your decision about what to share is yours alone. Please know that the STXBP1 community is here to support you and there should be no expectations that information be shared by families in trials.
If you choose to share any information, we encourage you to share only general information or tips like navigating travel This post from the Fragile X community provides more examples of general information. In the end, we all want the same thing - treatments that increase the quality of our STXer’s lives. The best way to make this happen is to help researchers get clear, accurate results so they can determine which treatments are really effective and which ones aren't.
Here is an additional resource from the Dravet community on sharing personal experiences. Dravet Syndrome is a different neurodevelopmental disorder which is a few years ahead of our community in genetic therapy clinical trials.