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Leveraging Patient-Reported Outcome Measures for Optimal Dose Selection in Early Phase Cancer Trials
Better characterizing dose response to optimize dose finding has been underlined by the FDA’s Project Optimus, which aims to reform how doses are selected in oncology clinical trials, with a particular focus on maximizing efficacy and optimizing safety and tolerability [5]. This led to their subsequent guidance on dose optimization for new cancer treatments [2].
JMIR Cancer 2025;11:e64611
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We designed a triple-masked pilot randomized controlled trial (RCT) that aims to examine the safety and tolerability of MPH and modafinil (aim 1) as well as to assess trends in changes in gross motor function and spasticity with the use of MPH and modafinil in children with CP (aim 2). In this placebo-controlled study, participants will be randomized into 3 groups that will receive MPH, modafinil, or placebo tablets.
JMIR Res Protoc 2024;13:e53728
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Yet, the research is limited on the feasibility, acceptability, safety, and tolerability of VCT-based treatments for perinatal individuals with depression, including home-visited mothers.
REST’s acceptability, safety, and tolerability were explored in 2 implementation-effectiveness hybrid pilot trials [16,21]. The first study included 13 home visited families (N=26 individuals) and a historical comparison group of 13 home visited depressed mothers who refused treatment [16].
JMIR Form Res 2023;7:e51824
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Still, most stated that they would still continue to seek alternative treatment for AKs in the future and would still be open to other topical treatments, especially if associated with a better safety and tolerability profile than 5-FU. Nonadherence to treatment is a major cause of treatment failure. Particularly, up to 50% of dermatology patients may be nonadherent to treatment regimens [16]. Ineffective communication about treatment-related AEs may predispose patients to poor adherence [16,17].
JMIR Dermatol 2023;6:e39988
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Reference 33: Sleeping at home: feasibility and tolerability of ambulatory polysomnography for use withtolerability
JMIR Mhealth Uhealth 2020;8(10):e20590
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