Discussions with the pharmaceutical/biotech industry as well as the investor community always begin the same way: How can we accurately test Rett patients to know if a potential medication is working? Are there one or two measures that the Rett clinical community agrees on and that are objective, reproducible and sensitive to change? Regrettably, the answer to that question is “no”. A quick survey of outcome measures for the trials listed on the clinicaltrial.gov website corroborates the situation.
The outcome measure issue is a serious knowledge gap and one that RSRT dedicated considerable time to in 2015 and will continue to do so this year. You’ll be hearing a lot about how we are tackling this topic in the months to come.
For now, we’d like your input with regards to an important pilot study we are funding aimed at measuring how Rett patients walk. The pilot study is a collaboration between Hassan Ghasemzadeh, the director of the Embedded and Pervasive Systems Lab (EPSL) at Washington State University, and Dr. Daniel Tarquinio, the director of the Rett clinic at Emory University in Atlanta.
The goal is to measure how kids walk over an extended period of time and in their own environment. Motion and pressure sensors will be embedded underneath the insole of sneakers that will allow data to be analyzed on a variety of parameters including stride length, gait speed, and cadence (rate at which a person walks, expressed in steps per minute). The objective of the study will be to measure gait before, during, and after a clinical trial to determine if a medication has an impact. Importantly, children who are unable to walk independently and require assistance could be measured as well. The study will outfit five pairs of sneakers of different sizes with sensors.
