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Leveraging Patient-Reported Outcome Measures for Optimal Dose Selection in Early Phase Cancer Trials

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].

Bill Byrom, Anthony Everhart, Paul Cordero, Chris Garratt, Tim Meyer

JMIR Cancer 2025;11:e64611

Psychostimulant Medications for Physical Function and Spasticity in Children With Cerebral Palsy: Protocol for a Randomized Controlled Trial

Psychostimulant Medications for Physical Function and Spasticity in Children With Cerebral Palsy: Protocol for a Randomized Controlled Trial

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.

Mansour Alotaibi, Anwar B Almutairi, Saleh Alhirsan, Afrah Alkazemi, Maha Alharbi, Naif Alrashdi, Ahmad Taqi, Bibi Alamiri, Laura Vogtle, Mohammed M Alqahtani

JMIR Res Protoc 2024;13:e53728

Video-Delivered Family Therapy for Perinatal Women With Depressive Symptoms and Family Conflict: Feasibility, Acceptability, Safety, and Tolerability Results From a Pilot Randomized Trial

Video-Delivered Family Therapy for Perinatal Women With Depressive Symptoms and Family Conflict: Feasibility, Acceptability, Safety, and Tolerability Results From a Pilot Randomized Trial

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].

Fallon Cluxton-Keller, Mark T Hegel, Craig L Donnelly, Martha L Bruce

JMIR Form Res 2023;7:e51824

Refusal of Retreatment With Topical 5-Fluorouracil Among Patients With Actinic Keratosis: Qualitative Analysis

Refusal of Retreatment With Topical 5-Fluorouracil Among Patients With Actinic Keratosis: Qualitative Analysis

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].

Rohan Singh, Sarah McCain, Steven R Feldman

JMIR Dermatol 2023;6:e39988