Fragile X syndrome clinical trials: exploring parental decision‐making

Abstract

Background The objective of this research was to understand parental proxy decision‐making for drug trial participation for children with Fragile X syndrome (FXS). Specifically, we aimed to capture preferences, motivations, influencing factors and barriers related to trial involvement among trial joiners and decliners and describe ease of trial decision‐making and decisional regret. Methods Interviews were conducted with parents from two groups: those who chose to enrol their child with FXS in a trial (N = 16; Joiners) and those who declined trial participation (N = 15; Decliners). Data were coded and interpreted through inductive content analysis. Results Prominent decisional factors included attitudes about medicating FXS symptoms, potential for direct benefit (primarily evaluated through the degree of match between target outcomes and child symptomatology and drug mechanism), logistical convenience and perceived risks of side effects. The ultimate motivation for participation was potential for direct benefit. None of the parents reported decisional regret, and ease of decision‐making ranged from easy to difficult for our participants. Conclusions Therapeutic optimism was high among those who elected participation. Parents may benefit from an explanation of the rationale behind chosen outcome variables and may be more interested in trials that target or measure as an exploratory outcome the symptoms they find most concerning. Our findings reinforce the need for future trials to reduce participant inconvenience. Our results contrast with what has previously been observed in parents of children with life‐threatening conditions; parents of children with FXS may be more trial risk averse and find trial decisions to be harder. Parents of children with FXS considering trials may benefit from a decisional intervention aimed at deliberating motivations and barriers.

Parent perceptions, beliefs, and fears around genetic treatments and cures for children with Angelman syndrome

“…For trials of genetic therapies, information pertaining to the genetic mechanism and trial evidence may be complex and difficult to understand. It is therefore imperative that parents are well informed when deciding whether to have their child participate in a genetic therapy by well‐informed health practitioners (D'Amanda et al, 2019; Inglis et al, 2014; Mikat‐Stevens et al, 2015) and that they are provided with accurate information about the potential benefits and consequences in a way that is genuinely understandable for a lay audience (Johannessen et al, 2016).…”

“…D'Amanda, Peay, Wheeler, Turbitt, and Biesecker (2019) interviewed parents of children with FXS ( n = 16) who had given or declined consent for their child to be in a therapeutic drug trial ( n = 15). Parents who included their children within drug trials felt that the trial would have a positive impact upon the disease mechanism of FXS, would directly target and benefit specific characteristics of their child, and was positive for the FXS community.…”

“…A consistent finding across these three qualitative studies is that parents want to make carefully considered decisions around treatment options for their children. For this to occur, the process, treatments, procedures, and outcomes must be carefully described and explored with parents (D'Amanda et al, 2019; Inglis et al, 2014; Wagner et al, 2019). Positive attitudes towards treatments were associated with parents wanting to cure the perceived difficulties associated with their child's syndrome (such as intellectual disability, health conditions, or anxiety) and to improve the health and quality of life for their child (Inglis et al, 2014) and with being willing to participate in clinical trials (D'Amanda et al, 2019; Reines et al, 2017).…”

“…For this to occur, the process, treatments, procedures, and outcomes must be carefully described and explored with parents (D'Amanda et al, 2019; Inglis et al, 2014; Wagner et al, 2019). Positive attitudes towards treatments were associated with parents wanting to cure the perceived difficulties associated with their child's syndrome (such as intellectual disability, health conditions, or anxiety) and to improve the health and quality of life for their child (Inglis et al, 2014) and with being willing to participate in clinical trials (D'Amanda et al, 2019; Reines et al, 2017). Although parents were motivated to reduce their child's difficulties, parents did not want to risk changing their child's personality or strengths (Inglis et al, 2014; Reines et al, 2017).…”

“…This complex relationship highlights the importance of future explorations using approaches that both collate basic data as well as allow for open responses to explore the beliefs, hopes, and fears of parents in more depth. D'Amanda, Peay, Wheeler, Turbitt, and Biesecker (2019) interviewed parents of children with FXS (n = 16) who had given or declined consent for their child to be in a therapeutic drug trial (n = 15). Parents who included their children within drug trials felt that the trial would have a positive impact upon the disease mechanism of FXS, would directly target and benefit specific characteristics of their child, and was positive for the FXS community.…”