What's a meaningful change for someone with amyotrophic lateral sclerosis (ALS) or his or her caregiver? A new, MDA-supported study seeks to answer that question.
It's believed that physical or psychosocial changes associated with a new treatment that may be "statistically significant" when clinical trial results are analyzed may not be the same as changes that people with ALS and their caregivers consider meaningful.
Pharmaceutical companies, regulatory agencies, insurers and other stakeholders in the ALS treatment development community would like to know what changes people with the disease consider meaningful, and the magnitude of change that would be required before someone would be willing to tolerate risks or side effects or incur costs associated with new therapies.
About the study
The MDA/ALS Clinical Research Network is supporting and recruiting participants for a five-center study to develop a scale of "clinically meaningful" changes from the perspective of people with ALS and their caregivers.
There are five locations for this MDA-supported study, all of which are sites of MDA/ALS Centers: Columbia University in New York City; Massachusetts General Hospital in Boston; Methodist Neurological Institute in Houston; California Pacific Medical Center in San Francisco; and Emory University in Atlanta.
How to participate
The investigators are seeking 100 patient-caregiver pairs, each of which will be asked to answer questionnaires either in person or over the phone four times during a six-month period. Participants with ALS must be 20-85 years old, have a family caregiver willing to participate, live near a study site, and meet other study criteria.
Contact Allison DiRienzo at the main coordinating center, Columbia University, at (212) 305-4746 or firstname.lastname@example.org, for more information.