Clinical Trials – what you need to know about outcome measures

So you guys have definitely published a lot and I'll post the link to the Project Inchstone website for everyone to see.

You work with many, many different groups.

Can you tell us who you're working with? And then also tell us your strategy.

Like what is it that you are specifically doing or trying to achieve in Project Inchstone? And then also why it's called Inchstone.

I guess all three of those things in one, right? So what do you hope to achieve? What are you doing and who are you working with?

Yeah, I'll start with where the name came from.

So the idea is that we are not necessarily measuring developmental milestones, but inchstones in developmental progress.

And for some of the individuals that we work with, they are moving forward developmentally, but at a much slower pace.

And if we are just asking families about progress, milestones, then we're missing some of the progress that they are making.

So those smaller inchstones or steps in development, which are different than typical development, but can be really, really meaningful and impactful for a family.

So that's where the name comes from.

We are

really a multidisciplinary team.

So the inchstone project was founded by Gabi Conacher and Jayetta Hecker.

And Gabi is mom to a son with SCN8A, which is the name of the gene or the genetic condition that he has.

And he has a DEE and he's profoundly impacted.

So the goal was really to develop, I think Gabi will tell the story about how she just felt so frustrated and defeated participating in clinical trials or doctor's developmental assessments where she's just circling no on rating forms or just you know, watching her kid not be able to complete the tasks that were being asked of him.

So the project was formed by them to really create or identify or develop assessments that would capture the skills that he has and the progress that he's making.

There are psychologists on the team.

There are also statisticians, psychometricians.

We also have some specialists in Speech and language and a teacher of the visually impaired who works with kids with cortical visual impairment.

So it really is a multidisciplinary team.

We're all bringing our different experiences and knowledge to address this complex problem that we're facing.

Tell me why, in your own words, why these inchstones are so important in some people and why we may be missing things if we only look at, say, the milestones.

Yeah, there's been some research now that's starting to converge around the fact that individuals who might be more severely to profoundly impacted, small change might be very meaningful to them.