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A clinical trial involves the study of the safety, efficacy and/or dosage regimen of a therapeutic intervention (such as a drug) in humans selected according to predetermined criteria of eligibility (such as a defined severity of a specific disease), who are observed for predefined evidence of favourable and unfavourable effects.
In this phase 1 trial, Hegde et al. treat 13 individuals with advanced sarcoma with lymphodepletion followed by HER2-specific CAR T cells, which were found to be safe and showed antitumor activity.
CAR-T cell therapies targeting BCMA have shown promising responses in patients with multiple myeloma (MM), however primary resistance and relapse are frequently observed. Here the authors report the results of a phase I//II study of bispecific CAR T-cells targeting BCMA and CD19 in relapsed/refractory MM.
Final results of two studies — whose preliminary data led to regulatory approval of the gene therapy exagamglogene autotemcel — describe highly effective treatment of sickle-cell disease and transfusion-dependent β-thalassemia.
The lay summary of trial results to be provided to participants should be written in plain language, use infographics and be concise — something that currently is almost never achieved.
A randomized controlled trial involving a telemedicine-based approach for the management of patients with acute coronary syndrome had several clinical benefits relative to standard of care.
An argument framework, grounded in the sciences of reasoning, provides an alternative to medicine’s measurement framework for evaluating and synthesizing evidence in healthcare.