• The University of Rochester Center for Health + Technology is seeking caregivers of children with spinal muscular atrophy (SMA) for an outcome measurement validation study.
• The study will involve taking a 20-minute survey twice, two weeks apart.
• Interested individuals should contact the study coordinators to learn more about participating.

The University of Rochester Center for Health + Technology is seeking caregivers of children with spinal muscular atrophy for a study to test and validate an outcome measurement tool that will be used in clinical trials. The research project is called the Spinal Muscular Atrophy Caregiver Reported Health-Index (SMACR-HI) Validation Study. Outcome measurement tools are used to assess the impact of a health care service or treatment on an individual.

The study is in the last phase of validating two versions of the SMACR-HI. One version is an outcome measurement tool for caregivers of any child under age 18, and the second is for caregivers of children ages 5 to 18. The validation process is focused on confirming the reliability of the tool.

What Is the SMACR-HI?

The SMACR-HI is similar to the Spinal Muscular Atrophy Health Index (SMA-HI), an outcome measurement tool for children over age 8 and adults. The SMA-HI is a patient-reported tool used to assess an individual’s experience of how SMA impacts their life.

The questions on the SMACR-HI "deal with the same scope of symptoms, but we wouldn’t ask a caregiver to say how much pain a child is in, or what their emotional status is,” Jennifer Weinstein, one of the study’s coordinators, explained on a phone call with mySMAteam. “It’s about things that can absolutely be observed from a caregiver perspective.”

Jamison Seabury, another of the study’s coordinators, explained that the SMA-HI and SMACR-HI were developed independently from completely separate data sets.

How Will the SMACR-HI Be Used?

“This outcome measure will be used in future clinical trials to allow therapies to be evaluated based on the caregiver perspective of how the child functions,” Seabury said.

The SMACR-HI and other similar instruments are “really designed to measure granular changes in health,” Seabury added.

Weinstein explained that the SMACR-HI can be another tool for assessing the benefit of a drug in clinical trials. In studies of new drugs, researchers identify a primary end point, or the main result researchers measure to determine if a new treatment is successful.

Sometimes a treatment or therapy doesn’t achieve the primary end point. However, it may have other benefits for those who take it. “Patients who participate in these trials have a whole range of other symptoms that are important to them in their daily life,” Weinstein said. “Perhaps the treatment didn’t assist with [the primary end point], but it assisted with self-care, eating, or bending over. [These improvements] are very impactful. However, they’re not being measured in the general efficacy of the drug.”

A caregiver- or patient-reported outcome tool like the SMACR-HI “is the way by which the patient's voice can be brought into the outcome measure. It can really bring a great deal more evidence to the actual benefit of the drug,” Weinstein noted.

In addition to the clinical trial setting, the SMACR-HI could be used by doctors for monitoring care and SMA disease progression over time.

Who Can Participate in the Study?

The SMACR-HI validation study is open to caregivers of children and teens 18 and younger who have SMA. Seabury explained that caregivers are generally parents of children with SMA, but that a home health aide or other adult family member who cares for the child may be eligible.

Study participants will take a 20-minute survey twice, two weeks apart. The survey will include personal questions about the participant and their family, as well as questions about how SMA impacts their quality of life, including mental and emotional wellness.

For more information about participating in the study, contact Jennifer Weinstein at jennifer.weinstein@chet.rochester.edu or (585) 419-5335; or Jamison Seabury at jamison.seabury@chet.rochester.edu or (585) 867-1461.

SMACR-HI Research Background

Dr. Chad Heatwole is the principal investigator of the SMACR-HI Validation Study. Dr. Heatwole serves as a professor of neurology and the associate director of the Center for Health + Technology at the University of Rochester. His research expertise is developing outcome measurement tools for pharmaceutical trials. Dr. Heatwole focuses on SMA, as well as other neuromuscular conditions. Dr. Heatwole was also involved in validating the SMA-HI.