Clinical trials articles from across Nature Portfolio

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.

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.

Risankizumab outperformed ustekinumab for the treatment of moderate to severe Crohn’s disease in patients refractory to anti-TNF therapy.

A large randomized controlled trial with cisgender women highlights the challenges of adherence to a daily oral preexposure prophylaxis regimen and demonstrates that twice-yearly injection of lenacapavir can maintain effective HIV prevention levels over 6 months.