Purpose

Digital therapeutics are a new class of interventions that are software driven and are intended to treat various conditions. We developed and evaluated a dichoptic digital therapeutic for amblyopia, a neurodevelopmental disorder for which current treatments may be limited by poor adherence and residual vision deficits.

Design

Randomized controlled trial.

Participants

One hundred five children 4 to 7 years of age with amblyopia were enrolled at 21 academic and community sites in the United States. Participants were randomized 1:1 to the treatment or comparison group, stratified by site.

Methods

We conducted a phase 3 randomized controlled trial to evaluate the safety and efficacy of a dichoptic digital therapeutic for amblyopia. Participants in the treatment group used the therapeutic at home for 1 hour per day, 6 days per week and wore glasses full-time. Participants in the comparison group continued wearing glasses full-time alone.

Main Outcome Measures

The primary efficacy outcome was change in amblyopic eye visual acuity (VA) from baseline at 12 weeks, and VA was measured by masked examiners. Safety was evaluated using the frequency and severity of study-related adverse events. Primary analyses were conducted using the intention-to-treat population.

Results

Between January 16, 2019, and January 15, 2020, 105 participants were enrolled; 51 were randomized to the treatment group and 54 were randomized to the comparison group. At 12 weeks, amblyopic eye VA improved by 1.8 lines (95% confidence interval [CI], 1.4–2.3 lines; n = 45) in the treatment group and by 0.8 lines (95% CI, 0.4–1.3 lines; n = 45) in the comparison group. At the planned interim analysis (adjusted α = 0.0193), the difference between groups was significant (1.0 lines; P = 0.0011; 96.14% CI, 0.33–1.63 lines) and the study was stopped early for success, according to the protocol. No serious adverse events were reported.

Conclusions

Our findings support the value of the therapeutic in clinical practice as an effective treatment. Future studies should evaluate the therapeutic compared with other methods and in additional patient populations.

中文翻译：

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弱视双视数字疗法的随机对照试验

目的

数字疗法是一类新的干预措施，由软件驱动，旨在治疗各种疾病。我们开发并评估了一种用于弱视的双视数字疗法，弱视是一种神经发育障碍，目前的治疗可能受到依从性差和残留视力缺陷的限制。

设计

随机对照试验。

参与者

105 名 4 至 7 岁的弱视儿童在美国的 21 个学术和社区网站登记。参与者以 1:1 的比例随机分配到治疗组或对照组，按地点分层。

方法

我们进行了一项 3 期随机对照试验，以评估双视数字治疗弱视的安全性和有效性。治疗组的参与者每天在家中使用治疗剂 1 小时，每周 6 天，并全职戴眼镜。对照组的参与者继续独自全职戴眼镜。

主要观察指标

主要疗效结果是 12 周时弱视眼视力 (VA) 从基线的变化，并且 VA 由蒙面检查者测量。使用研究相关不良事件的频率和严重程度评估安全性。使用意向治疗人群进行初​​步分析。

结果

2019 年 1 月 16 日至 2020 年 1 月 15 日期间，共有 105 名参与者参加；51人被随机分配到治疗组，54人被随机分配到对照组。在 12 周时，治疗组的弱视眼 VA 改善了 1.8 线（95% 置信区间 [CI]，1.4-2.3 线；n = 45）和 0.8 线（95% CI，0.4-1.3 线；n = 45） ) 在对照组中。根据方案，在计划的中期分析中（调整后的 α = 0.0193），组间差异显着（1.0 线；P  = 0.0011；96.14% CI，0.33-1.63 线），并且研究因成功而提前停止。没有报告严重的不良事件。

结论

我们的研究结果支持治疗在临床实践中作为有效治疗的价值。未来的研究应与其他方法和其他患者群体相比，评估治疗方法。