Articles tagged with Clinical Studies
The company's ATA-200 gene therapy has shown safety, pharmacodynamics, and efficacy results in the first patients treated, offering hope for children with LGMD-R5. The therapy delivers a normal copy of the γ-sarcoglycan gene and has been awarded Orphan Drug Designation in the US and Europe.
The Alliance for Clinical Trials in Oncology is spotlighting new trials for colorectal cancer in March, focusing on early detection methods and treatments for treatment delays and loss of appetite. The trials aim to improve patient outcomes, with several enrolling patients with newly diagnosed colon or rectal cancer.
A new clinical study aims to improve communication between patients and families about genetic risks of colorectal cancer. The trial will compare two methods of sharing genetic test results with close relatives, with the goal of learning which approach helps more family members get necessary genetic testing.
A new study by Cleveland Clinic and Dyania Health demonstrates the potential of large language models in accurately identifying patients who could benefit from clinical research. The AI system reviewed 1,476 patients and identified 46 as potential matches, resulting in a more diverse patient population.
Researchers at Adelaide University have discovered a naturally occurring protein that can protect against sepsis by reducing inflammation. The protein, corticosteroid-binding globulin (CBG), has been shown to reduce relative mortality in an animal model with septic shock by over 70 per cent.
The European Medicines Agency has granted a positive opinion to Acoziborole Winthrop as a single-dose oral treatment for both early- and advanced-stage gambiense sleeping sickness in adults and adolescents. The medicine, co-developed by DNDi and Sanofi, could provide a significant advance over current therapies.
Researchers found that circulating tumor DNA can predict metastatic risk in patients treated with bladder-sparing treatment for muscle-invasive bladder cancer. The absence of ctDNA predicted favorable outcomes regardless of bladder removal status.
Daily aspirin use offers no quick or reliable way to prevent bowel cancer in the general population, increasing risk of serious bleeding. However, it may have potential long-term benefits for people at high genetic risk of colorectal cancer.
Approximately 40 cancer patients will receive LMP744 for five consecutive days, with biological analyses conducted on brain tissues before and after treatment. If results are favorable, treatment will continue for 12 cycles to evaluate parameters such as progression-free survival and overall survival.
The Barshop Institute will conduct the VITAL-H trial, evaluating the repurposing of FDA-approved medications to delay age-related health decline in generally healthy middle-aged adults. The trial aims to preserve everyday abilities during a critical window of midlife aging.
The Milken Institute and Ann Theodore Foundation have launched a new funding program, ATF-SIM, to support a Phase 2 clinical trial assessing the effectiveness of mTOR inhibitors in treating cutaneous sarcoidosis. The grant will award up to $575,000 to researchers to design and conduct the trial.
The University of California San Diego School of Medicine and UC San Diego Health are now part of the NICHD Maternal-Fetal Care Network, expanding access to research on pregnancy and newborn health. Clinical trials will focus on maternal mortality, prematurity birth rates, and infant mortality rates.
A new Cochrane review suggests that intermittent fasting may not be an effective strategy for weight loss in overweight or obese adults. The study found no meaningful difference in weight loss between intermittent fasting and traditional dietary advice or doing nothing, highlighting the need for more research into this area.
A new study found that tuberculosis is a major cause of deadly sepsis among people living with HIV, particularly in Africa. Early treatment of TB has been shown to significantly reduce sepsis deaths, with a 23% reduction in mortality rate.
A new review introduces a chronological and probabilistic model to explain the recurrence of autoimmune skin diseases like vitiligo. The model proposes that full disease manifestation is the outcome of genetic susceptibility and a series of environmental or internal biological hits.
Researchers discovered that ATG triggers rapid clotting and inflammation in the liver by activating a complement activation system and TGF-β pathway, leading to severe side effects. The study used human organoid models and imaging analysis to reveal the biphasic mechanism of ATG-induced liver injury.
The American College of Cardiology's Global Quality Solutions program now allows international hospitals to use approved internal data to meet accreditation requirements. This update enables hospitals to internally collect and submit aggregated data directly into ACC Accreditation tools, streamlining the accreditation process.
The study validates the use of SOFA-2 in sepsis care and research, demonstrating its ability to identify clinically meaningful organ dysfunction. The findings suggest that incorporating immune markers may not improve predictive performance, highlighting the biological complexity of sepsis.
A blind patient partially recovered natural vision through electrical stimulation of the visual cortex, independent of the implant. The recovery was sustained over time and remained even after the device was removed, suggesting individual factors may have contributed to this outcome.
OYE-101, a novel intravenous caffeine formulation, aims to accelerate recovery from general anesthesia and deep sedation in adults. The FDA-cleared treatment also seeks to address opioid-induced respiratory depression and related overdose situations.
Researchers at Johns Hopkins Kimmel Cancer Center have developed a novel liquid biopsy approach to identify early-stage cancers by measuring DNA methylation patterns. The Epigenetic Instability Index (EII) successfully distinguished patients with early-stage lung and breast cancers from healthy individuals, showing promise in detecting...
A national clinical trial found that oxybutynin significantly reduced hot flash frequency and quality of life for men undergoing hormone therapy for prostate cancer. The study showed substantial improvements in hot flash symptoms, often within the first week of treatment.
The Mental Health Goals programme aims to transform mental health research infrastructure with a £50 million government-funded initiative. King's College London, Cardiff University, and University of Oxford will co-lead two workstreams to develop the world's largest dataset for depression and improve links between research and industry.
The phase III PATINA study demonstrated that adding CDK4/6 inhibitor palbociclib to standard maintenance therapy significantly prolongs progression-free survival by more than 15 months compared to standard therapy alone. The study found a meaningful improvement in median PFS with no new signals.
Cancer cells tap into the nervous system's power grid by forming synaptic contacts with nerve cells, promoting tumor growth and spread. Venkataramani's research aims to repurpose the drug perampanel for glioblastoma treatment and develop gene therapy approaches to disconnect tumors from the nervous system.
A new review of over 100 clinical trials found that 13 common labor induction methods are similarly effective, with low-dose vaginal misoprostol being the most commonly used and safest. However, some methods showed better success rates for specific outcomes, such as oxytocin with amniotomy for achieving vaginal birth within 24 hours.
A new study from Nationwide Children's Hospital finds that similar to older children and adults, about one fourth of children under six years old who experience a concussion will develop prolonged symptoms. Younger children are more likely to sustain brain injuries due to their size differences and weaker muscles.
A new AI-powered model predicts patient survival and risk stratification for spinal metastasis, allowing for more informed treatment decisions. The model achieved high predictive accuracy using large-scale clinical data from Japanese patients.
Insilico Medicine has received investigational new drug (IND) clearance from the US FDA for ISM8969, an orally available NLRP3 inhibitor targeting inflammation and neurodegenerative disorders. The Phase I clinical trial plans to evaluate the safety, tolerability, and pharmacokinetics of ISM8969 in healthy volunteers.
The ISSCR has released a comprehensive roadmap for developing pluripotent stem cell-derived therapies, outlining critical scientific, manufacturing, and regulatory guidance. The Best Practices document provides a risk-based, end-to-end development strategy to support informed decision-making across seven essential areas.
The Phase 2 ANDROMEDA trial combines two types of PSMA-targeted radiopharmaceuticals with stereotactic body radiotherapy to delay progression and minimize side effects in patients with oligorecurrent prostate cancer. Researchers aim to determine which approach provides the most durable cancer control.
A major U.S. clinical trial has uncovered a genetic factor that may inform how to optimize the dosing of abiraterone, a widely used hormone treatment for advanced prostate cancer. Researchers found that men who carry a specific version of the gene SULT2A1 clear abiraterone from their bodies more slowly, which could affect how well it w...
A new clinical trial, PAGODA, seeks to minimize treatment interruptions and help patients complete their chemotherapy as planned. The trial will test a structured plan to guide doctors in making small, proactive changes to chemotherapy doses to prevent treatment delays.
Insilico Medicine's Phase IIa clinical trial assesses Garutadustat's safety and efficacy in ulcerative colitis. The novel PHD inhibitor has shown favorable safety and tolerability profiles in completed Phase I studies.
A single-dose oral cholera vaccine called PanChol has completed a phase 1 clinical trial with positive results, offering hope for combating the devastating disease globally. The vaccine was developed by Mass General Brigham and shows promise in preventing severe vomiting and diarrhea caused by Vibrio cholerae bacteria.
A recent study published in Gut journal revealed that Streptococcus anginosus produces methionine metabolites, which significantly contribute to the development of gastric cancer. The research opens new paths for microbiota-targeted prevention strategies.
A new clinical trial, RECIPROCAL, seeks to optimize targeted radiation therapy for men with advanced prostate cancer by adjusting treatment based on individual PSA levels. The trial aims to minimize side effects while preserving survival benefits.
The Baveno VII consensus guidelines have reshaped portal hypertension management, focusing on early intervention and noninvasive diagnostics. Nonselective beta-blockers and transjugular intrahepatic portosystemic shunt (TIPS) are recommended for clinically significant portal hypertension diagnosis.
A new study by Case Western Reserve University and University Hospitals found that financial factors are the strongest predictors of participation in cancer research studies. The researchers recommend addressing practical challenges like transportation, childcare, and lost wages to make clinical trials more inclusive.
The new framework addresses inefficiencies in current PET system accreditation methods, introducing a contrast recovery coefficient-based system for reproducibility and reliability. Universal adoption will provide advantages for stakeholders, enabling trusted data comparison across the globe.
A blood test may help doctors identify which patients with colon cancer can benefit from anti-inflammatory medication and chemotherapy after surgery. The test measures circulating tumor DNA levels, and high-risk patients who test positive see improved survival rates when taking celecoxib with chemotherapy.
A study found that only 6% of FDA-approved drugs reflect the US racial and ethnic makeup, with a decline in Black and Hispanic enrollment between 2021 and 2023. The researchers recommend setting diversity goals at preclinical stages and collecting biological samples to understand how people's bodies react to medications.
A study from Rangsit University shows that a simple bypass protocol guided by mean transit time can improve symptoms in 80% of patients with chronic cerebral occlusion or moyamoya disease. The protocol uses non-stress testing to evaluate cerebrovascular reserve and predict postoperative improvement.
A patient with refractory synovial sarcoma was the first in Arizona to receive TECELRA, a genetically modified autologous T cell immunotherapy. The treatment showed early signs of tumor shrinkage and has been shown to benefit over 40% of patients with similar cancer types.
The ISSCR symposium highlights new clinical trial results and emerging technologies advancing PSC-derived cell therapies. Key findings include breakthrough approaches for treating drug-resistant epilepsy and geographic atrophy, as well as durable remission in type 1 diabetes.
The International Linked Clinical Trials Program aims to change that by supporting and facilitating clinical trials of potentially disease-modifying treatments. Over 6,800 people with Parkinson's have participated in these trials, and approximately 40% of prioritized drugs have been approved for clinical trials.
A new study presented at ASH 2025 suggests that a virtual program focusing on diet and exercise can help reduce the severity of side effects associated with lymphoma treatment. The program, LIFE-L, showed significant improvements in symptoms such as anxiety, depression, pain, fatigue, and constipation among participants.
The program provides financial aid and unique clinical experiences to medical students, encouraging them to become community physicians. UCF College of Medicine hopes the program can be used as a national model to address the nation's primary care physician shortage.
Researchers at the University of Plymouth will receive a £2.8 million funding boost to accelerate new treatments for low-grade brain tumors. The center aims to deepen understanding and translate knowledge into life-changing therapies.
A Cochrane review found no significant effect of calcium supplementation on pre-eclampsia, contradicting long-held assumptions. The review excluded small and unreliable studies that previously suggested benefits of calcium supplementation.
Researchers argue that widely used proxies for aging may conflate genuine modifications with simpler effects on physiology. Targeting specific pathologies can extend lifespan without slowing aging process. Historically, increased human lifespan was primarily driven by reduced infectious disease mortality, not a fundamental slowing of a...
Two new Cochrane reviews show that HPV vaccines are effective in preventing cervical cancer and pre-cancerous changes, especially when given to young people before they are exposed to the virus. Girls vaccinated before the age of 16 were found to be 80% less likely to develop cervical cancer.
A new clinical trial enrolls patients at the University of Cincinnati to study the effects of extended tranexamic acid dosing on recovery outcomes for young athletes after ACL reconstruction. The trial aims to test whether this approach can reduce pain, swelling and promote earlier movement in patients.
Linda Bulone, a Queens Cancer Center Oncology nurse, received the 2025 Alliance Clinical Research Professionals Research Integrity Service Excellence (CRP RISE) Award. She has significantly increased clinical trial participation, expanded early screening efforts, and supported reductions in late-stage diagnoses.
A study published in Journal of Translational Medicine found that successful root canal treatment can reduce inflammation linked to heart disease and improve levels of blood sugar and cholesterol. The researchers discovered a link between monitoring blood metabolic markers after dental treatment, which could help assess recovery and risk.
A clinical trial at UCI Health has tested a new CAR T-cell therapy for lupus patients. The treatment showed significant reversal of symptoms in the first trial participant, offering hope for future treatments.
A new Yale study found that only 24% of medicines tested in clinical trials received market authorization, highlighting a significant 'test it but don't sell it' problem. The study highlights the need for collective effort from pharma companies, countries, and other stakeholders to improve post-trial access to life-saving medicines.
A recent study found that changes in a single gene can cause mental illnesses, with the GRIN2A gene identified as a key culprit. Variants of this gene are associated with schizophrenia and other mental disorders, which can manifest in childhood or adolescence rather than adulthood.
Researchers at UC Riverside and City of Hope have developed a novel Pin1 degrading compound that suppresses pancreatic cancer peritoneal metastases. The treatment targets not only cancer cells but also tumor-supporting cells, potentially overcoming treatment resistance.
The Phase 3 study demonstrated the safety and non-inferiority of EuTYPH-C Inj.® Multi-dose compared to a World Health Organization (WHO) prequalified comparator TCV, Typbar TCV®. The vaccine was shown to be well-tolerated across all age groups with no serious adverse events.