G.P.303: Influences on parental decision making about participation in Duchenne and Becker muscular dystrophy clinical trials

Abstract

Introduction: Parents’ decisions about enrolling children in Duchenne/Becker muscular dystrophy (DBMD) clinical trials (CTs) may be influenced by hopes and expectations, which when unrealistic may challenge informed consent. We explore influences on decision-making. Methods: The study team employed a community-based participatory research approach. Parents of children in CTs in U.S. or Canada were recruited for an online survey through Parent Project Muscular Dystrophy and clinics. Participants viewed a series of benefit and worry statements and rated each statement’s influence on decision-making, and how much they expected and hoped or worried about each. Results: Among the first 61 participants, CT decision-making was influenced by potential altruistic and individual benefits: learning generalizable information (97% slightly to strongly agree), CT resulting in a drug that works (82%), leading to better future for other children (82%), improving quality-of-life for their child (82%), and parent doing everything to help their child (82%). CT worries most affecting decision-making were: child would be bothered by side effects (39%), child’s eligibility for a competing trial (36%), and child not liking the trial (29%). Expectation and hope ratings were highly correlated with ratings of factors that influenced decision-making ( r = 0.5–0.8, p