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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 the phase 2 NICHE-3 trial, patients with locally advanced mismatch repair-deficient colon cancer who were treated with neoadjuvant anti-PD1 and anti-LAG3 agents showed high rates of pathological responses, requiring validation in larger trials.
This phase I/II clinical trial was designed to assess safety and efficacy of gene therapy for Bothnia dystrophy, an inherited retinal degeneration leading to blindness. Kvanta et al. show interim results from 12 patients with meaningfully improved visual function in dim light.
Li et al. perform a phase 2 single-arm clinical trial of neoadjuvant chemotherapy plus checkpoint blockade in participants with resectable muscle-invasive bladder cancer and conduct genomic and transcriptomic profiling to describe molecular subtypes.
This Perspective calls for inclusion of patients with MASLD and measurement of liver outcomes in cardio–kidney–metabolic trials, when data suggest mechanistically plausible benefits and clinical safety—and outlines considerations for trial design and regulatory approval.
Post-approval evidence generation is essential for high-quality clinical care and should be a shared priority for clinicians, health systems, payors, and the medical products industry, as well as the FDA and federal agencies.
In people undergoing medical abortion after 12 weeks of pregnancy, those who took the first dose of misoprostol at home were less likely to require overnight hospitalization.
Researchers developed an AI-enabled, battery-operated tool that can be operated by clinicians with no sonography experience — and that measures gestational age as accurately as high-specification ultrasound.
In a digital-only study, people were more likely to take a home test if prompted to do so on the basis of self-reported symptoms rather than by wearable sensor data, with implications for public health responses and pandemic planning.