(159) A Qualitative Examination of Participation in the SIMPLIFY Treatment Withdrawal Trial

Background: In the era of effective modulator therapy, several clinical trials evaluating the impact of withdrawal of other daily CF therapies have been initiated. We sought to understand the reasons people with CF (PwCF) and their caregivers had for enrolling in a randomized treatment withdrawal study, as well as to explore their experiences of study participation.

Methods: The QUEST (Qualitative Understanding of Experiences in the SIMPLIFY Trial) study is an ongoing qualitative interview study enrolling participants after they complete participation in the CFF-sponsored SIMPLIFY trial, a randomized control trial evaluating short-term withdrawal of hypertonic saline or dornase alfa therapy among individuals on a stable modulator therapy regimen. Participants in QUEST were recruited from 39 SIMPLIFY sites and included adults and teenagers with CF, as well as caregivers of the teenage participants. Two interviews were conducted remotely via Zoom (audio only) or telephone and the first interview occurred within 4 months of completion of their SIMPLIFY trial participation. The interviews were recorded and transcribed. Analysis of the transcripts was carried out using codes developed inductively by the research team. Coding was performed independently by two coders. This analysis focuses on interview 1, as this explored reasons for participation in SIMPLIFY.

Results: 106 participants completed interview 1 (64 adults, 21 teens, and 21 caregivers). Among participants with CF, median age was 24.8 years, and 53% were female. Participants talked about joining SIMPLIFY out of curiosity, a desire to help others or to advance scientific knowledge, in addition to finding out if they personally could stop a therapy. However, some noted they joined for the compensation, to reduce the burden on their family and/or friends, or because their care team suggested they join. Caregivers also noted fostering responsibility in their adolescent child as a reason for study participation. The decision to participate in SIMPLIFY was taken independently by the adult participants but many noted that they consulted family and/or their care team. When specifically considering experiences during the SIMPLIFY trial, all participants in QUEST accepted their randomized assignment. Those who expressed disappointment in their study arm assignment were most often assigned to maintain therapy. When asked about joining future withdrawal studies, participants expressed a willingness to do so but noted that they would need to be ‘healthy’ and not feel that they were putting their health at risk. Notably several PwCF were emphatic that they would not join a study about withdrawing modulator therapy. Overall, participants expressed hope and optimism around the possibility of withdrawing other therapies in the future.

Conclusions: Participation by PwCF in the SIMPLIFY treatment withdrawal study was driven by factors both personal and community-minded, often in the context of increased optimism for the future. Future withdrawal studies in CF should consider highlighting these factors to foster research participation from PwCF.

Acknowledgements: Funding provided by CF Foundation: QUEST20PE0, SAWICK14PE1, RIEKER15PEO

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