An expansion in the availability of clinical drug trials for genetic neurodevelopmental conditions is underway. Delineating patient priorities is key to the success of drug development and clinical trial design. There is a lack of evidence about parent decision-making in the context of clinical drug trials for genetic neurodevelopmental conditions. We assessed parents’ priorities when making a decision whether to enroll their child with fragile X syndrome (FXS) in a clinical drug trial. An online survey included a best–worst scaling method for parents to prioritize motivating and discouraging factors for child enrollment. Parents were recruited through the National Fragile X Foundation and FRAXA. Sequential best–worst with conditional logit analysis was used to determine how parents prioritize motivating and discouraging factors about trial enrollment decisions. Respondents (N = 354) were largely biological mothers (83%) of an individual with FXS who ranged in age from under 5 to over 21 years. The highest motivating factor was a trial to test a drug targeting the underlying FXS mechanism (coeff = 3.28, p < 0.001), followed by the potential of the drug to help many people (coeff = 3.03, p < 0.001). Respondents rated requirement of blood draws (coeff = −3.09, p < 0.001), loss of access to the drug post trial (coeff = −3.01, p < 0.001), and drug side effects (coeff = −2.96, p < 0.001) as most discouraging. The priorities defined by parents can be incorporated into evidence-based trial design and execution to enhance the enrollment process.

针对遗传性神经发育疾病的临床药物试验的可用性正在扩大。描述患者的优先级是药物开发和临床试验设计成功的关键。在针对遗传性神经发育疾病的临床药物试验的背景下，缺乏关于父母决策的证据。在决定是否让患有脆性 X 综合征 (FXS) 的孩子参加临床药物试验时，我们评估了父母的优先事项。一项在线调查包括一种最佳-最差缩放方法，供家长优先考虑孩子入学的激励因素和阻碍因素。父母是通过 National Fragile X Foundation 和 FRAXA 招募的。顺序最佳 - 最差条件逻辑分析用于确定父母如何优先考虑有关试验注册决定的激励和劝阻因素。受访者（N  = 354) 大部分是 FXS 患者的亲生母亲 (83%)，年龄从 5 岁以下到 21 岁以上不等。最高的激励因素是测试针对潜在 FXS 机制的药物的试验（coeff = 3.28，p  < 0.001），其次是药物帮助许多人的潜力（coeff = 3.03，p  <  0.001）。受访者对抽血要求 (coeff = −3.09, p  < 0.001)、试验后无法获得药物 (coeff = −3.01, p  < 0.001) 和药物副作用 (coeff = −2.96, p  < 0.001)进行了评分作为最令人沮丧的。家长确定的优先事项可以纳入循证试验设计和执行，以加强注册过程。