Articles tagged with Clinical Trials
A three-year-old girl has been successfully treated with the first-ever FDA-approved gene therapy treatment for AADC deficiency, a rare inherited neurological disorder. The treatment, Kebilidi, is expected to take 2-6 months to show measurable impact and has already shown signs of increased energy and happiness.
A new study published in The Annals of Thoracic Surgery found that over 6% of patients experienced early hemodynamic valve deterioration just one year after undergoing TAVI. Smaller implanted valve sizes were significantly associated with the development of HVD, while larger valves appeared to be protective.
In a phase 1 clinical trial, the engineered antibody improved a class of drugs that had struggled to deliver on its early promise. Six patients experienced systemic tumor reduction, including two complete responses, with minimal side effects.
A Phase 1 human clinical trial has commenced at Griffith University to test the efficacy and safety of a revolutionary new treatment using olfactory ensheathing cells. The trial aims to repair and regenerate nerves in patients with chronic spinal cord injury, with intensive rehabilitation before and after transplantation.
The BREATHE-3 clinical trial evaluates a novel bronchoscopic device to open airways and release trapped air in patients with severe emphysema. The study will assess the safety and efficacy of the Apreo BREATHE Airway Scaffold, an implantable device granted FDA Breakthrough Device Designation.
Researchers have found a way to rearrange atoms in a new generation of malaria drugs to make them more soluble, maintaining their effectiveness against drug-resistant parasites. This breakthrough could lead to an effective successor to artemisinin-based combination therapy.
A three-year follow-up study of a personalized program of cognitive functional therapy (CFT) found clinically significant improvements in pain-related activity and pain intensity compared to usual care. The treatment produced large benefits that were sustained over time, offering potential for major human and economic impact.
Local-stage colorectal cancer incidence among US adults ages 45-49 increased by 50% between 2021 and 2022, with a steep rise in 2019-2022. Screening rates also increased during this period, driven by recommendations to start screening at age 45.
Researchers at Kyoto University have discovered a novel analgesic called ADRIANA, which targets the α2B-adrenoceptor and exerts its effect without causing cardiovascular instability. The compound has shown highly promising results in non-clinical studies and early clinical trials.
Researchers conducted a randomized clinical trial to compare low- and high-dose methylprednisolone with azithromycin for treating severe Mycoplasma pneumoniae pneumonia. The study found that lower dose regimen of glucocorticoids is sufficient, equally effective, and safer than the high dose regimen.
The EFFECT Trial aims to evaluate the effectiveness of early fibrinogen replacement in trauma patients, with a potential to redefine resuscitation practices and save lives. The trial is a major collaboration between CU Anschutz and Octapharma, enrolling 800 participants across top-tier trauma centers nationwide.
Researchers develop machine learning models to analyze sparse digital footprints and forecast depressive relapses or manic episodes with clinical-level accuracy. The approach uses GPS-derived social withdrawal and erratic typing patterns to predict bipolar episodes 24 hours in advance.
A coordinated approach is needed to address the scientific value of trial representation of the intended use population. The study aims to provide a strategic framework to ensure scientific validity by operationalizing broad representation at all levels.
A new study published in JAMA Internal Medicine found that commercially available UV-C light appliances reduced rates of viral respiratory infections by more than 12% in aged care facilities. The technology is easily installed and cost-effective, making it a transformative solution for protecting residents from emerging pathogens.
Genethon is launching a Phase 3 clinical trial in Europe for its low-dose microdystrophin gene therapy GNT0004, targeting boys aged 6 to 10 with retained walking ability. The trial aims to demonstrate efficacy and tolerance of the treatment.
The US POINTER trial found that a structured lifestyle intervention of regular exercise, adherence to the MIND diet and social engagement had greater benefits on global cognition compared to an unstructured intervention. The study showed significant cognitive benefit after two years in older adults at elevated risk of Alzheimer disease.
Researchers from FAU's Schmidt College of Medicine recommend using high-potency statins as the primary pharmacologic approach in treating and preventing cardiovascular disease. Lifestyle changes, such as regular physical activity and a healthy diet, are also crucial in reducing cardiovascular risk.
VCU Massey Comprehensive Cancer Center is the first US site to enroll patients in a global Phase 2a clinical trial of PTX-100, a small molecule inhibitor for cutaneous T-cell lymphoma. The trial aims to evaluate the efficacy and safety of PTX-100 in patients with relapsed or refractory disease.
Allergic responses to tattoo ink can lead to severe symptoms like urticaria, angioedema, and granulomatous reactions. Laser therapy can worsen these conditions, leading to hypertrophic scars or keloids. Alternative treatments like corticosteroids and surgical excision are recommended.
Breakthroughs in treating age-related macular degeneration (AMD) include FDA-approved pegcetacoplan and avacincaptad pegol, targeting the complement system to slow geographic atrophy progression. Anti-VEGF therapies like faricimab also offer extended dosing intervals, reducing injection frequency.
Researchers used an AI model to reassess a completed clinical trial for an Alzheimer's disease drug, finding it slowed cognitive decline by 46% in early-stage patients. The AI model predicts patient progression and allows for more precise selection of trial participants, potentially reducing the cost of developing new medicines.
A pilot clinical trial has been initiated to test the efficacy of alpelisib in treating Dent disease 2, a rare genetic disorder affecting kidney function. The trial aims to improve abnormal kidney function and potentially provide a breakthrough treatment for patients with life-threatening complications.
A cohort study found that GLP-1RAs like semaglutide and tirzepatide lower risk of dementia, stroke, and all-cause mortality in adults with type 2 diabetes and obesity. The findings suggest potential neuroprotective benefits beyond glycemic control.
A new study published in the Journal of Hepatology shows that durvalumab plus GemCis significantly improves overall survival for patients with advanced biliary tract cancer. The three-year follow-up analysis found that over twice as many participants treated with durvalumab plus GemCis remained alive compared to those treated with a pl...
A new treatment method using microglia replacement has shown promising results in halting the progression of genetic neurological disease ALSP in both mice and human individuals. The treatment, developed at Fudan University, successfully replaced mutated microglia with healthy ones, improving neurological function and extending life ex...
A new study found that a nontargeted screening approach can identify more patients with hepatitis C virus infections than traditional targeted screening methods. The results highlight the need for improved treatment models, as patients who went from diagnosis to sustained virologic response increased significantly.
This randomized clinical trial found that bedtime dosing of antihypertensive medication improves nocturnal blood pressure control and circadian rhythm without compromising daytime or 24-hour blood pressure. The study suggests potential advantages of bedtime administration for patients with hypertension.
A clinical trial found that women experience stronger subjective effects of acute alcohol intake, such as drunkenness and sedation, compared to men. The study adjusted doses to achieve identical blood alcohol concentrations for both sexes, revealing that women's effects were more intense.
A new multi-institutional clinical trial will test zipalertinib, an investigational drug targeting specific EGFR mutations in early-stage non-small cell lung cancer. The goal is to prevent recurrence and improve long-term outcomes for patients with limited treatment options.
A genetically engineered herpes simplex virus, RP1, shows promise in treating advanced melanoma when combined with immunotherapy. In a phase 1-2 clinical trial, one-third of patients experienced significant tumor shrinkage or disappearance, offering hope to those with untreatable cancer.
A randomized clinical trial found that cannabidiol (CBD) administration led to liver enzyme level elevations in 5.6% of participants and potential drug-induced liver injury in 4.9%. Hepatic enzymes returned to normal within weeks after discontinuation. Further research is needed on CBD's long-term effects and safety.
Intra-arterial tenecteplase significantly reduces the risk of symptomatic intracranial hemorrhage and mortality in patients with acute large vessel occlusion. The treatment also improves neurological outcomes at 90 days without increasing complications.
The study proposes a modified ResUNet approach for automated liver segmentation from CT images, achieving high precision and accuracy. The model outperforms traditional methods, demonstrating stable performance in cross-dataset tests.
Researchers developed an innovative FENCY ligation technique combined with preoperative embolization to remove giant plexiform neurofibromas, reducing risk of complications and improving recovery time. The study results in significant functional improvement and high patient satisfaction rates.
Researchers at the Wyss Institute have identified vorinostat as a promising treatment for Rett Syndrome using an AI-driven drug discovery process and innovative disease modeling. The findings demonstrate disease-modifying abilities across multiple tissues, offering hope for a potentially curative treatment.
A new study highlights major hurdles for multinational clinical trials in Europe, including differences in timelines and processing times between countries. Researchers found that the UK made significant gains in efficiency during the pandemic, while non-UK countries lagged behind.
A comprehensive study protocol has been developed to investigate pregnancy-related stroke risk factors and long-term implications for mothers and their children. The nationwide multicenter study aims to identify key risk factors associated with ASPP and assess its impact on offspring.
Researchers have discovered an oral drug called MA-5 that can improve both heart and muscle problems in patients with Barth syndrome, a rare genetic disorder. The drug boosts cellular energy production by up to 50% and protects cells from oxidative stress-induced death, addressing the underlying cause of the disease.
Researchers at Mass General Brigham found a novel strategy to help patients control their disease, increasing eligibility for stem cell transplants. The combination of duvelisib and romidepsin was effective, tolerable, and safe for patients with relapsed/refractory peripheral and cutaneous T-cell lymphomas.
A landmark national study in China identifies the most effective treatment strategies for unruptured brain aneurysms, analyzing over 874,000 clinical entries and 42,000 radiological scans. The trial aims to guide patient-specific treatment strategies and inform healthcare planning in China's aging population.
A Flinders University study found that providing consistent support and access to nicotine replacement therapies can help people in recovery quit smoking. The trial showed that both vapes and combination therapy were effective, with around 10% of participants abstaining from smoking at the nine-month follow-up.
A collaborative study from the University of Cologne has discovered the key role of mitochondrial protein AIFM1 in regulating cellular energy metabolism. The research found that AIFM1 interacts with AK2A to maintain energy homeostasis, and visualized its complex structure using cryo-electron microscopy.
A phase 3 trial demonstrated the efficacy of selumetinib in reducing tumor size and alleviating pain in adults with neurofibromatosis type 1. The treatment was found to be effective in patients who received it from day one, as well as those who started it later, with significant reductions in tumor size and pain reported.
Researchers at the University of Missouri have developed a new imaging probe that can help surgeons accurately identify and remove aggressive brain tumors during operations. The tool, known as FA-ICG, uses near-infrared light to highlight tumor cells from within.
A phase 1 clinical study will assess the safety and efficacy of JBZ-001 in patients with AML. The study is supported by a $3.4 million grant from the National Cancer Institute and aims to improve efficiency for the therapeutic pipeline.
Researchers at University of Cologne have discovered a previously unknown mechanism that explains why many patients with aggressive B-cell lymphoma don't respond to CAR-T cell therapy. Combining this innovative treatment with CSF1R inhibitors improves its effectiveness, according to the study published in Cancer Cell.
Insilico Medicine has completed the first-in-patient dosing of ISM3412, a novel MAT2A inhibitor with potential as a best-in-class treatment for locally advanced and metastatic solid tumors. The Phase 1 study aims to evaluate safety, tolerability, and preliminary anti-tumor efficacy of ISM3412.
A new international study identified two distinct genetic signatures for complex forms of Alzheimer's disease, with one primarily driven by apolipoprotein E and the other involving a combination of 75 additional genetic variants. This shared biological mechanism is largely consistent across global populations.
The UNC Lineberger Comprehensive Cancer Center has developed an adaptive clinical trial for metastatic breast cancer, leveraging $28 million in funding from the Advanced Research Projects Agency for Health (ARPA-H). The study aims to adapt treatment plans in near real-time using tumor biopsies, blood samples, and biomarkers.
The BathMat clinical trial evaluates an innovative inflatable pillow designed to make moving intensive care unit patients safer, faster, and less labour-intensive for NHS staff. The trials aim to reduce staff time required for repositioning proned patients and improve patient healthcare outcomes.
Researchers at UC Davis developed a brain-computer interface that translates neural activity into speech in real time. The technology allows individuals with ALS to communicate more naturally and inclusively, with 60% of synthesized words intelligible to listeners.
Researchers have discovered that nicotinamide riboside supplementation significantly improves NAD+ levels, reducing arterial stiffness, skin ulcer area, and kidney dysfunction. This breakthrough offers new hope for patients with Werner syndrome who lack effective treatment options.
The SHARE guidelines offer a systematic checklist to report sham acupuncture control in clinical trials, covering elements of sham acupuncture and related contextual factors. This comprehensive reporting promotes accurate interpretation of trial results and enhances the quality of sham acupuncture-controlled clinical trials.
Long-term data from the Phase 1/2 study of zigakibart demonstrate sustained proteinuria remission, stable kidney function, and a reassuring safety profile in patients with IgA nephropathy. The treatment also led to reductions in serum immunoglobulins, consistent with APRIL pathway inhibition.
The annual Alzheimer's disease drug development pipeline report, led by Dr. Jeffrey Cummings, highlights an increase in active clinical trials and promising drugs, offering reason for optimism. The report assesses 138 drugs currently being studied and notes the growing importance of biomarkers in clinical trials.
The clinical trial demonstrated that the vaccine was safe and fully attenuated, causing no malaria infections in adults. The vaccine has the potential to achieve unprecedented potency and is designed to align with WHO's goals for malaria elimination.
The Phase IIa trial of Rentosertib showed encouraging clinical data, with patients receiving the treatment experiencing significant improvements in lung function compared to placebo. The study also validated the biological mechanism of TNIK inhibition using exploratory biomarkers analyses.
The S2302 Pragmatica-Lung trial found that the investigational combination of ramucirumab and pembrolizumab did not extend overall survival compared to standard-of-care treatments. The study enrolled a highly representative population of patients, establishing it as a paradigm-shifting model for large randomized studies.
A recent randomized controlled trial found that a group-based intervention fostering communication and coping skills in a culturally tailored way improved outcomes for Latina breast cancer survivors and their caregivers. The study, published in CANCER journal, demonstrated high engagement among participants and clinically significant i...
A global clinical trial led by Irish cancer specialist Prof. Patrick Forde shows that immunotherapy with chemotherapy before lung cancer surgery reduces the risk of cancer returning and improves long-term survival. The study found that patients who received immunotherapy plus chemotherapy had improved outcomes compared to those who rec...