Smashing Barriers Helps Recruit More Study Participants

By Rivkah Lewin

Public access to genetic testing has enabled more than 26 million to explore if they’re at risk for genetic diseases (1).  No one is thrilled to know that they’re more likely to get a rare illness, but the upside of finding out is that some inherited maladies are suitable for therapies that target the specific genetic mutation that’s the culprit.  Indeed, 95% of participants in the Valor-PD  (Virtual Assessment of LRRK2 Carriers to Optimize Research in Parkinson’s Disease) study would like to join a Parkinson’s disease prevention trial.  

Study researchers wanted to identify a group of candidates who would be appropriate to participate in future clinical trials of gene-directed therapies for Parkinson’s.  However, the gene that causes the illness is a rare variant, so “It would be very difficult to recruit the number of people we have in the study from a single site if it was a traditional in-person study,” explains Ruth Schneider, M.D., Department of Neurology, University of Rochester Medical Center, who heads the trial.  Because the gene is so uncommon and the three-year study required hundreds of participants, the 277 people who joined had to be recruited from all over the U.S.  

Recruitment focused on Ashkenazic Jews, a population with a high prevalence of the relevant G2019S mutation, the most common determinant of Parkinson’s identified as of March 2017 (2).  Most people in the study don’t have Parkinson’s, but whether they have it or not, travel is a major barrier that makes people nix joining a study, and may be especially arduous for disabled individuals.  Traditionally, research studies are conducted at academic sites and many potential participants are not close to the research locations so they won’t even consider joining.  That’s one reason why designers of Valor-PD demolished the travel hurdle and made the study entirely remote.  

An added benefit of going virtual is reduced costs for infrastructure and personnel.  Though no research study has done a head-to-head comparison of remote vs. multi-site setups, says Schneider, she suspects Valor-PD’s virtual model is cheaper than a traditional multi-site setup.  That would’ve required having sites all over the country, personnel at each site, and a core coordinating group directing the study across all locations.

Another strategy of the study designed to plough down obstacles to participation was that the real-time video visits, where study team members meet with participants, are relatively short and can typically be completed in about an hour.  Researchers can accomplish their goals faster by making it possible for participants to do assessments outside of the video visit.  They can answer some surveys at their convenience, from home, work, or wherever they choose  before and after the video visits.

They can even do the “smell test,” a “scratch & sniff” assessment developed by the University of Pennsylvania, that tells researchers about the individual’s sense of smell from the comfort of their home.  Why a smell test?  “Parkinson’s disease is a progressive, neurodegenerative disorder,” Schneider observes.  “Classically, when people think of Parkinson’s, they think of tremor, slowness of movement, and stiffness.  But it actually is much more than that.”  In addition to those motor symptoms, patients may experience non-motor difficulties, including loss of sense of smell, cognitive issues, constipation, anxiety, depression, lightheadedness, and pain.  So the study’s assessments include a full range of symptoms people may have, including loss of sense of smell.  Valor-PD is a beacon of hope for sufferers, though, as it can help accelerate development of new treatments for the malady. 

 1. More than 26 million people have taken an at-home ancestry test | MIT Technology Review

2.  LRRK2 G2019S Mutation: Prevalence and Clinical Features in Moroccans with Parkinson’s Disease – PubMed (nih.gov)