Research on the trials that test new treatments in patients. Covers trial design, respiratory and cancer trials, screening tools, and treatment breakthroughs.
Elinzanetant, a selective neurokinin-1,3 receptor antagonist, demonstrated significant reductions in vasomotor symptoms frequency and severity vs placebo in postmenopausal individuals with moderate to severe VMS. The treatment also improved sleep disturbances and menopause-related quality of life, with a favorable safety profile.
A clinical study confirmed the safety of aceneuramic acid in treating distal myopathy with rimmed vacuoles (GNE myopathy), a rare disease causing progressive muscle weakness. The treatment, developed for an ultra-orphan drug, showed efficacy and was approved by Japan's Ministry of Health, Labour and Welfare.
A disproportionality analysis of World Health Organization data revealed a signal of semaglutide-associated suicidal ideation, particularly among patients with co-reported antidepressant use. This finding warrants urgent clarification and highlights the need for further research on the safety profile of semaglutide.
Scalable telehealth mindfulness-based interventions show promise in treating chronic pain by improving function and reducing symptoms. The low-resource approach could accelerate the adoption of nonpharmacological pain treatment in healthcare systems.
A study examining tecovirimat's effectiveness on mpox in the DRC found the drug safe but showed no significant impact on lesion duration. The trial, sponsored by the NIH and co-led with the DRC, resulted in lower mortality rates among participants compared to reported cases.
A new vaccine has shown to protect mothers from malaria during pregnancy and for up to two transmission seasons without booster doses. The vaccine was well-tolerated and safe for both the mothers and their offspring, with no differences in adverse events compared to placebo.
A malaria vaccine candidate has demonstrated sustained protection against parasite infection and clinical malaria over two years without a booster dose. The vaccine significantly protected women from malaria in pregnancy, offering new strategies to prevent the disease in vulnerable populations.
Researchers developed a new brain-computer interface that translates brain signals into speech with up to 97% accuracy, enabling a man with amyotrophic lateral sclerosis (ALS) to communicate with friends and family. The system was tested in real-time conversations with continuous updates, achieving high word accuracy rates.
A clinical trial shows that JAK inhibitors improve autoimmune conditions such as alopecia areata, atopic dermatitis, and psoriasis in people with Down syndrome. The study also observed improvements in arthritis and decreased biomarkers of autoimmune thyroid disease.
A clinical trial in Denmark found that offering women further screening opportunities during breast cancer screenings significantly increased their participation and enabled early diagnosis of other cancers. The study used a cluster-randomized crossover design and involved over 27,000 participants.
Researchers uncovered how combining immunotherapies targeting PD1 and LAG3 enhances CD8+ T cell responses, leading to improved cancer-killing prowess and enhanced survival rates. The studies also revealed that relatlimab is not inert and can be combined with other immunotherapies to improve responses.
A recent study by Neda Akhavan found that baked potatoes can help lower blood sugar and improve body composition in adults with Type 2 diabetes. The study, which involved 24 participants, showed a modest decrease in fasting blood glucose levels and improvements in waist circumference and resting heart rate.
A new study found significant improvements in aerobic fitness after 12 weeks of high-intensity interval training sessions compared to traditional, moderate exercise. Researchers suggest this time-efficient approach can be safely and effectively implemented in clinical practice for stroke survivors.
A large clinical food trial will be conducted to test the effects of a combination of probiotics and prebiotics on bone health in women aged 60 years and above. The study aims to develop potentially safe and inexpensive dietary interventions for osteopenia and osteoporosis.
A UofL cardiologist is leading a Phase II clinical trial of a new stem cell therapy for heart failure, the first in the US to use intravenous delivery. The trial aims to determine whether one or multiple doses of mesenchymal stem cells improve health and quality of life for patients.
A clinical study in Birmingham has found that nipocalimab can improve the survival rate of unborn babies with rare fetal anaemia. The majority of pregnant patients who received nipocalimab gave birth at or after 32 weeks without needing an intrauterine transfusion, resulting in a perinatal survival rate of 92%.
Researchers analyzed data from 63,700 patients in five clinical trials to understand the impact of clonal hematopoiesis of indeterminate potential (CHIP) on heart disease. They found that patients with CHIP had a 30% higher risk of initial heart attacks but not recurrent events.
A clinical trial found that fish oil supplements slowed neuronal integrity breakdown in older adults with the APOE4 gene variant, a risk factor for Alzheimer's disease. The study showed no benefit for all older adults, but suggested omega-3 fatty acids may be useful for prevention.
Researchers at Wake Forest University School of Medicine are studying the effectiveness of non-pharmacological treatments for migraine, which affects nearly half of those with the condition and leads to significant disability. The five-year study aims to identify therapies that target long-term efficacy and safety.
A dual-chamber wireless pacemaker demonstrates reliable performance over three months, synchronizing contraction of the upper and lower heart chambers 98% of the time. The device continues to work well even when patients engage in activities or have elevated heart rates.
The PD GENEration study has found that 13% of participants have a genetic form of Parkinson's disease, significantly higher than previous estimates. The study, which reached its goal of 15,000 participants ahead of schedule, provides insights into the genetics of the disease and its potential for precision medicine.
Tumors with mutations in ERCC2, ATM, or RB1 genes were more likely to be eliminated by cisplatin-based chemotherapy. The study found a correlation between these gene mutations and complete pathologic response to neoadjuvant chemotherapy.
A recent study published in ESC Heart Failure found that kidney dysfunction can be used as a criterion to stratify patients requiring implantable defibrillators. The research identified two key independent predictors for the risk of sudden cardiac death: left ventricular ejection fraction and estimated glomerular filtration rate.
Researchers found that combining plasma p-tau217 and Aβ42/40 levels could predict early brain Aβ accumulation in people with subthreshold Aβ accumulation. These biomarkers may help screen participants for primary prevention trials.
A randomized clinical trial found that posterior foraminotomy provides outcomes comparable to those of the more commonly performed anterior cervical discectomy. The two-year surgical success rate was 81% in patients assigned to posterior surgery, similar to 74% in the anterior group.
Research from Bentley University shows that biotechnology companies can maintain current levels of new drug approvals in face of global revenue reductions. Strategic distribution of cost reductions and continued investment in early-stage development by biotech firms sustain pipeline, contradicting industry claims.
A new study found that two Medicaid policies can interact to increase oncology clinical trial enrollment among Black and Hispanic patients. Medicaid expansion under the ACA alone did not improve diversity, but states with pre-existing mandates requiring coverage of clinical trial costs saw a significant increase.
The Wake Forest University School of Medicine has been awarded a $28.2 million grant from the National Institutes of Health to accelerate the translation of scientific discoveries. The grant will support initiatives such as rapid response to health emergencies and the recruitment of diverse academic learning health system workforce.
A clinical trial found that fecal microbiota transplants can boost the effectiveness of immunotherapy in a range of gastrointestinal cancers. The study identified specific bacterial strains associated with better or worse responses, and showed promise for improving treatment outcomes.
Researchers found that personalized text messages effectively increased physical activity among patients after significant heart events, with Apple Watch users showing a 10% increase in step count. However, the benefits later tapered off as patients became accustomed to the messages.
Researchers assessed botulinumtoxin-A treatment to improve walking capacity in children with cerebral palsy. The trial found some evidence of reduced calf pain and delayed improvement in energy cost, but no significant benefit for overall walking ease.
Researchers at KAUST have developed NanoRanger, an accurate and rapid method for genetically diagnosing Mendelian genetic disorders. This breakthrough enables diagnosis in just 12 minutes, providing a detailed picture of the genomic disorder.
The A4 study, a large clinical trial of pre-symptomatic Alzheimer’s disease, has yielded key insights into the condition. Researchers have collected extensive data on brain scans, blood samples, genetic information and cognitive tests from over 7,500 participants.
The PrEPVacc HIV vaccine trial found no significant reduction in HIV infections among participants. The study reported more infections in the vaccine arms compared to the placebo groups, but the results are inconclusive due to wide statistical confidence intervals.
A randomized clinical trial found that overground robot-assisted gait training using a wearable robot significantly improved gross motor function and gait pattern in children with cerebral palsy. The study suggests that this type of assistance may complement standard rehabilitation by providing adequate support.
A phase II clinical trial found that combining sEphB4-HSA with pembrolizumab improved outcomes in HPV-negative, EphrinB2-positive head and neck squamous cell carcinoma patients. The combination showed a favorable toxicity profile and activity, particularly among those with HNSCC.
A randomized crossover trial found that a joint-AI model improved clinical diagnosis, but future trials are warranted to confirm its effectiveness. The AI model demonstrated positive human-AI interaction, suggesting its potential to facilitate accurate diagnoses.
Researchers found that subretinal adeno-associated virus 8 injections trigger proinflammatory T-cell responses, but co-injection of immunodominant peptides can modulate the immune system. This study suggests a new approach to AAV gene therapy for retinal diseases.
Researchers from Radboud University Medical Center discuss the potential of using early on-treatment circulating tumor DNA measurements as a response assessment for metastatic castration-resistant prostate cancer. The detection of ctDNA at baseline and 4-weeks after treatment initiation can predict response durability to first-line ARPIs.
A new approach in vaccine research, adaptive platform trials (APTs), offers a flexible adjustment of study design during ongoing clinical trials. This allows for quick adaptation to new developments and enables the recruitment of large numbers of participants across different countries.
The study establishes criteria for identifying extranodal extension on imaging, allowing radiologists to make accurate calls and provide patients with less invasive treatments. This shift towards practice change has the potential to reduce side effects and improve outcomes for patients with HPV-associated head and neck cancers.
Researchers discuss HER2 in breast cancer, highlighting the prognostic and predictive factors. They found an independent positive prognostic effect of HER2-low compared to HER2-zero in early breast cancer, challenging the long-held view that HER2-negative tumors do not benefit from trastuzumab-based therapy.
A new framework assesses research integrity in clinical guidelines, excluding untrustworthy studies and prioritizing transparency. The RIGID framework has been applied to several international guidelines, removing problematic studies that posed a risk to patient care.
A study published in Nature Medicine found that off-the-shelf wearable trackers can monitor the response to two treatments for atrial fibrillation and heart failure. The devices provided clinically useful information similar to in-person hospital assessments, with a neural network helping to analyze missing data.
The study found that massage therapy has moderate-certainty evidence for some conditions but rare conclusive evidence of its superiority over other treatments. The review highlights the need for more research in this area.
A short-term reduction in leisure-time screen media use within families was found to positively affect psychological symptoms of children and adolescents. This effect was particularly notable in mitigating internalizing behavioral issues and enhancing prosocial behavior.
A study found that drivers who received weekly feedback on their phone use and a cash incentive of up to $50 used their phones less than those given no incentive or feedback. The intervention also led to a 21% decrease in handheld phone usage when drivers were told they would lose money if they engaged in phone use while driving.
A Phase 2 clinical trial demonstrated the effectiveness of OXO-001 in increasing embryo implantation, pregnancy and live birth rates among infertile women. The study showed significant improvements in biochemical, clinical and ongoing pregnancy rates, with a notable increase in live birth rates.
A web-based cognitive behavioral self-help intervention effectively decreases eating disorder symptoms in individuals with bulimia nervosa, reducing illness-related burden. The digital intervention has potential as a complement to established treatments for bulimia nervosa.
Immunotherapy has emerged as a promising approach in treating metastatic colorectal cancer, especially for patients with dMMR/MSI-H tumors. The use of immune checkpoint inhibitors has shown favorable outcomes in clinical trials, including improved progression-free survival and disease control rates.
The NIH is sponsoring a Phase 1 trial of an experimental nasal COVID-19 vaccine that may offer enhanced protection against emerging variants. The trial will enroll 60 adult participants and assess the safety and immune response of the vaccine candidate, MPV/S-2P.
Scientists have developed a new immunotherapy that can identify and fight cancer cells in patients with Merkel cell carcinoma. The treatment involves stimulating the immune system's T cells against specific elements of the virus involved in cancer formation.
A groundbreaking clinical trial has shown that gene therapy can halt the progression of spastic paraplegia type 50 (SPG50) in a young boy. The treatment, delivered via spinal fluid, eliminated serious side effects and showed potential signs of improvement.
A study of 4,782 Sri Lankan children aged 10-18 with dengue found that obese individuals were more than twice as likely to be hospitalized due to the disease. The research highlights the potential consequences of rising childhood obesity rates on public health.
A study found that patients with treatment-refractory cancers who received protocol exemptions to participate in a large basket/umbrella oncology trial experienced similar rates of clinical benefit and adverse events as patients who participated in the trial without waivers. The results suggest that broadening eligibility criteria for ...
A recent review of studies on heart failure patients suggests that reducing salt intake has no clinical benefits and may even be counterproductive. Moderate to strict salt restriction was linked with improved quality of life but not mortality or hospitalization rates among patients with heart failure.
Two studies published in The New England Journal of Medicine show mixed results in treating EoE. Dupilumab is found to be effective in children under 12, while benralizumab fails to provide significant relief for patients. These findings challenge the long-held assumption that eosinophils are the primary cause of the disease.
Lenacapavir has shown 100% efficacy as PrEP among cisgender women in a Phase 3 clinical trial. The twice-yearly injection offers improved feasibility of PrEP for those experiencing barriers to daily pill-taking. NIAID looks forward to additional data and regulatory decisions.
A randomized clinical trial found that combining exercise with glucagon-like peptide-1 receptor agonist (GLP-1RA) treatment was the most effective weight loss strategy while preserving bone health. In contrast, GLP-1RA treatment alone reduced bone mineral density at clinically relevant sites.
A new AI model has been developed to improve clinical trial recruitment for eye disease, specifically Geographic Atrophy. The system identified almost twice as many candidates and with higher precision compared to conventional approaches, showing promise for overcoming a major obstacle in GA clinical trials.