Experiences of families with a child, adolescent, or young adult with neurofibromatosis type 1 and plexiform neurofibroma evaluated for clinical trials participation at the National Cancer Institute.

With increasing knowledge of the molecular pathways contributing to the progression of neurofibromatosis type 1 (NF1)-related plexiform neurofibroma (PN), the number of clinical trials for PNs has increased. However, little is known about the experiences of families with children with NF1 participating in clinical trials. A 47-item anonymous survey, designed to assess experiences in research, was sent to parents with a child with NF1 and PNs who was evaluated at the National Cancer Institute (NCI) for a clinical trial. Sixty-four (85%) parents completed the paper or online questionnaire. Fifty-nine percent of the children were male; 44% were 11 years or older upon enrollment. Most parents reported understanding the purpose of the study, possible side effects, and their right to withdraw. Of respondents whose child had participated in a placebo-controlled trial, 100% said they understood the reason for the placebo at least somewhat. Seventy-four percent felt that study participation helped their child; most would "definitely" or "probably" participate in a future study, including a placebo-controlled trial, and particularly those assessing cognitive functioning or pain. Overall satisfaction with participation was high and correlated with fewer transportation problems, fewer study-related financial difficulties, and fewer school problems for the child (ps<.05). Most parents of children with NF1 who participated in research at the NCI reported a positive impact and would participate in future studies. Respondents identified several areas for improvement. This report may help plan future studies to optimize experiences of children and their families enrolled in clinical trials.