Abstract
The primary goal of clinical trials is to advance our understanding of human disease, yet participants may have the potential to see direct benefits on their condition. Novel therapeutic clinical trials specific to Duchenne muscular dystrophy (DMD), a life-limiting condition, have begun to show promising results, but inclusion criteria for these trials often require ambulation, a criterion that the majority of older boys with DMD do not meet. The impact of ineligibility for eagerly anticipated clinical trials has never been examined. \r The purpose of this study was to explore the feelings of parent caregivers of boys with DMD that are ineligible for novel therapeutic clinical trials for DMD. We interviewed seven parents of non-ambulatory boys with DMD using a semi-structured interview guide to assess the impact of exclusion from clinical trials on parental hopefulness, expectations for the future, and continued interest in clinical research. To find common and divergent themes, we used software to identify and code themes that emerged from the interview transcripts.\r Four common themes emerged: (1) Ambulation represents a barrier to research participation, and parents are often frustrated by their exclusion due to this barrier. (2) Parents conveyed resignation to their son’s condition, his ineligibility, and the limitations regarding therapies to be developed during his lifetime. (3) Parents’ active interest in clinical research opportunities waned. (4) Parents still expressed hope for their sons, often related to the concept of emotional well being, but also to the possibility of a cure or slowing the progression of his condition. These results highlight how parents rationalize being left out of clinical trials while maintaining hope. These parents’ hope likely represents healthy coping with chronic disease. Genetic counselors and other providers should continue discussing research with older boys with DMD.