By: Lauren Schmitt, PhD, Chief Science Officer, PMSF
Date: December 22, 2025
How Families Can Help Protect the Path to New Treatments
Clinical trials are essential to advancing treatments for Phelan-McDermid syndrome. They represent years of scientific work, significant commitment from families, and real hope for meaningful progress. Because so much is at stake, it is critically important that trial data are collected and interpreted without bias.
That is why families participating in clinical trials are strongly discouraged from sharing personal trial experiences with other participants, extended family and friends, or on social media while a study is ongoing.
This guidance can feel counterintuitive. Our community values connection, shared experience, and mutual support. But when it comes to clinical trials, even well-intentioned sharing can have serious and lasting consequences.
Why caution during trials is so important
Clinical trials are designed to answer one central question: does a treatment truly work, and is it safe? To do that, studies rely on carefully controlled conditions that minimize bias.
When families share information about their child’s experience during a trial, including side effects, perceived improvements, or lack of change, it can unintentionally interfere with those controls.
This is not a hypothetical concern. In other rare disease communities, including Fragile X syndrome, sharing clinical trial details has directly contributed to trials being compromised. In some cases, promising studies were negatively impacted or shut down entirely, even when a drug appeared to be helping patients.
When a trial fails because of compromised data, no one has access to its findings. This can have a devastating and long-lasting impact, not only on the current participants but also on the entire community that may miss out on a potential therapeutic access.
How sharing can compromise a trial
Even informal or private conversations can affect study outcomes in several ways:
Unblinding the study
Many trials are blinded, meaning families and researchers do not know who is receiving the investigational treatment versus placebo. Discussing side effects or perceived benefits can lead families to guess group assignment, undermining the scientific validity of the study.
Bias and placebo effects
Reading or hearing about other families’ experiences can subconsciously influence how caregivers observe and report changes. This can inflate placebo effects or alter symptom ratings.
Gaming entry criteria
In past trials, families shared details about how to qualify or how scoring worked. This led to inflated baseline scores, distorted data, and ultimately the loss of drugs that may have been beneficial.
Regulatory consequences
If trial data are compromised, regulatory agencies may reject the results — even if the treatment truly works — because the data can no longer be trusted.
What families should not share during an active trial
To protect the integrity of clinical research, families should avoid posting or discussing:
❌ Side effects (real or suspected)
❌ Behavioral, developmental, or communication changes
❌ Improvements or lack of improvement
❌ Videos or anecdotes showing new skills (e.g., new words, pointing, learning letters)
❌ Comparisons between children in the trial
❌ Information about how to qualify for a study or how scoring works
❌ Details of study surveys or tasks
This applies to public social media posts, private groups, and direct messages with other trial participants.
What is okay to share
Families still deserve connection and support. Sharing non-clinical, logistical information does not affect trial integrity and is encouraged. This includes:
✅ Travel tips, lodging, or parking information
✅ Hospital navigation and practical setup tips
✅ General praise for trial sites, coordinators, or staff
✅ Encouragement for other families (without discussing outcomes)
These types of sharing help families support one another without influencing study data.
Who to talk to about concerns
Any concerns about side effects, changes, or questions related to your child’s participation in a clinical trial should be discussed with the study team, such as the trial coordinator or principal investigator. These professionals are responsible for monitoring safety and are trained to collect and interpret information in a way that protects both the participant and the integrity of the study.
Families may also consult their loved one’s regular medical or care team, especially when that team is separate from the study site. A child’s primary care provider or specialists are often involved in the decision to participate in a trial and may help assess or manage side effects alongside the study team.
If a participant experiences urgent or severe symptoms, families should seek immediate medical care (including emergency services) and notify the study team as soon as possible.
Families should not seek validation or advice about trial effects from other trial participants, either privately or online.
A shared responsibility
Clinical trials are a partnership between families, researchers, clinicians, and regulators. Families play an essential role — not only by participating, but by helping protect the integrity of the data.
Choosing not to share trial experiences during a study is not about silence or isolation. It is an act of advocacy for the entire community. Protecting the science today helps ensure that effective treatments can be proven, approved, and ultimately made available to everyone affected by Phelan-McDermid syndrome.
We are deeply grateful to the families who participate in clinical research and who help safeguard its future through thoughtful, responsible engagement.