Clinical Trials - Science topic

Background Research on pilot studies is scarce, despite their importance for the development of effective clinical trials. There is a lack of work addressing the process, limitations and strategies derived from these studies. This gap is even more evident in research studying targeted populations, such as victims of intimate partner violence (IPV),...

Many cancer therapies fail in clinical trials despite showing potent efficacy in preclinical studies. One of the key reasons is the adopted preclinical models cannot recapitulate the complex tumor microenvironment...

El objetivo del presente estudio fue revisar sistemáticamente la evidencia disponible sobre la efectividad del rodillo de espuma vibratorio (VFR) frente al rodillo de espuma convencional (FR) en el dolor, rendimiento deportivo y rango de movimiento (ROM) en personas físicamente activas. Se realizó una búsqueda sistemática en las bases de datos Medl...

Objective To evaluate the efficacy of 2 alcohol-based hand preparations (61% weight/weight ethanol and 1% chlorhexidine gluconate [Avagard; 3M] and 85% weight/weight ethanol [Sterillium; BODE Chemie GmbH]) in reducing bacterial colony count with the glove-juice method. We hypothesized that there would be no difference between hand preparations imme...

Objective: To conduct a systematic review of motivational interviewing (MI) integrity in randomized controlled trials and to conduct a meta-analysis on the effect of MI-informed interventions compared to control conditions on gambling behavior and gambling disorder symptom severity at posttreatment and follow-up. Method: Five databases were searche...

Aim To investigate how caregivers of children with developmental and epileptic encephalopathy and severe developmental impairments describe meaningful change for functional domains and why it is important. Method This was a convergent mixed‐methods design study. A survey was completed by 267 parents of children aged 12 months or older. For priorit...

The global health burden of sepsis is immense, characterized by significant loss of life and high healthcare costs. Traditional Chinese medicine (TCM), with its over two millennia of clinical practice in China, has gained attention as a potential adjunctive approach for sepsis. Here, we evaluated TCM applications in sepsis management, highlighting...

INTRODUCTION There is a need to understand the clinical meaningfulness of symptom score changes in treatment trials of dementia‐related agitation. We estimated minimal clinically important differences (MCIDs) for commonly employed agitation scales and contextualized their clinical application. METHODS We employed anchor‐ and distribution‐based app...

Objective Although remimazolam tosilate (RT) has demonstrated efficacy in elderly gastroscopy, its optimum dose for gastroscopy in elderly patients of varying ages is still unclear. The study aimed to determine the median effective dose (ED 50 ) and 95% effective dose (ED 95 ) of RT to inhibit the gastroscopy reaction in patients aged 65–74 years a...

Background. Recurrent primary glomerulonephritis is a frequent and severe disease that represents the second or third leading cause of graft loss. Objective. The purpose of this study is to address the rates of recurrence for all types of glomerulonephritis, detailing their characteristics and the treatments adopted. Methods: The authors collected...

Metabolic dysfunction–associated steatotic liver disease (MASLD) diagnosis and management have evolved rapidly alongside the increasing prevalence of obesity and related complications. Hepatology has expanded its focus beyond late-stage cirrhosis and portal hypertension to earlier, complex MASLD cases in younger patients, necessitating closer colla...

Budesonide orodispersible tablets (BOT) have been shown to be efficacious for eosinophilic oesophagitis (EoE) in clinical trials. This study evaluated the real‐world effectiveness of BOT in a retrospective cohort of 94 patients across six tertiary centres. 61% of patients achieved histological remission and 39% clinicohistological remission followi...

Blinatumomab is a CD19 × CD3‐directed bispecific T‐cell engager that has become an essential backbone of acute B lymphoblastic leukaemia (BCP‐ALL) treatment. It is used in relapsed/refractory disease, minimal residual disease (MRD), as well as in first‐line treatment. Blinatumomab is particularly effective in MRD‐positive BCP‐ALL, inducing a high r...

Background Direct laryngoscopy (DL) is widely recognized as the most commonly used method for tracheal intubation. However, growing evidence highlights the increasingly prominent role of video laryngoscopy (VL) in the management of difficult airways. This study aimed to determine the most effective medical education method to equip medical students...

Background In recent years, therapeutic preparations using patient-derived tissues have emerged as commercially approved regenerative medicine products for expanding treatment possibilities for patients with no other treatment options. Autologous cell-processed products, derived from the tissue of the patient, typically exhibit variability in raw m...

Aims To investigate the potential pharmacokinetic interactions of montmorillonite powder or loperamide on pyrotinib. Methods This study was a single-center, open-label, single-dose, fixed-sequence clinical trial conducted with healthy volunteers. The participants were divided into two groups (A and B), each consisting of 18 subjects. Both groups r...

Objective This research is a quasi-experimental study conducted on male students from six elementary schools ranging from third to sixth grade in three regions of Shahrood city from March 2023 to February 2024. This study aimed to designing, implementing and evaluating a health-oriented self-learning game with a problem-solving approach in order to...

The first successful kidney transplant in December 1954 between the Herrick brothers ushered in a new field of medicine. Over the almost seventy years, thousands of lives have been saved and patient survival has improved. There is one area of kidney transplant patient care that has been overlooked. Patient quality and ability to participate in life...

Introduction: The ancient art of varmam, attributed to Siddhars, focuses on harnessing subtle energy to optimize bodily functions. Disruption of these functions due to imbalances in varmam points can lead to various diseases. Kateegraha, characterized by stiffness and pain in the lumbo-sacral region, is considered a manifestation of vaata imbalance...

Background Gene editing can produce irreversible permanent changes to the genetic material at predetermined sequences, avoiding random integration, which is the major drawback of classical gene therapy. The technology has invaded all approaches of genetic engineering and biotechnology with versatile applications in agriculture, industry, and medici...

Characterizing subtle cognitive changes in preclinical Alzheimer’s disease (AD) is difficult using traditional neuropsychological assessments. Remote and unsupervised digital assessments can improve scalability, measurement reliability, and ecological validity, enabling the capture of subtle changes. We evaluate such tools for use in preclinical AD...

Large-language models (LLMs) show promise for clinical note information extraction, but deployment challenges include high computational costs and privacy concerns. We used synthetic data distillation to fine-tune smaller, open-source LLMs to achieve performance comparable to larger models while enabling local hardware deployment or reduced cloud c...

DNA methylation is associated with various diseases and aging; thus, longitudinal and repeated assessments of methylation patterns are crucial for revealing the mechanisms of disease onset and identifying factors associated with aging. The presence of batch effects influences the analysis of DNA methylation array data. Since existing methods for co...

A prospective, single-arm, open-label Phase III clinical trial was conducted across multiple centers in China from April 27, 2020, to June 15, 2021, to assess the efficacy and safety of 10% intravenous immunoglobulin (IVIg) in treating adult immune thrombocytopenic purpura (ITP). Within 7 days of treatment initiation, the 10% IVIg group exhibited a...

Among the more than 90 identified genetic risk loci for late-onset Alzheimer′s disease (AD) and related dementias, the apolipoprotein E gene (APOE) ϵ2/ϵ3/ϵ4 polymorphism remains the longstanding benchmark for genetic disease risk with a consistently large effect across studies. Despite this massive signal, the exact mechanisms for how ϵ4 increases...

Impact The study documents significant changes in skin pigmentation among Kenyan newborns over their first month of life, with significant decreases in reflectance at wavelengths critical for transcutaneous diagnostic devices. This research provides the first quantitative documentation of optical-light characteristics in African newborn skin using...

Pantheism – All is God – is the hidden doctrine behind many reported psychedelic-occasioned experiences, both in the literature and in clinical trials, yet its meaning is little understood in the clinical and therapeutic spheres. This essay therefore seeks to remedy this deficit by offering a fresh outline of Pantheism via the exploration of its et...

Background: Real-world data (RWD) is essential in head and neck cancer (HNC) research, offering insights into outcomes among diverse, comorbid patients often underrepresented in clinical trials. Methods: We developed RT-HaND-C, a multi-source clinical dataset integrating structured EHR data, unstructured data extracted using an AI-driven NLP tool,...

Type 2 diabetes (T2D) poses a substantial global health burden. In 2023, we published an overview on the remission of T2D. Since then, additional long-term data has emerged regarding the outcomes associated with T2D remission, highlighting the importance of revisiting this topic. This article synthesizes findings from the landmark Diabetes Remissio...

CAR-NK cells offer a promising immunotherapy approach, addressing key CAR-T cell limitations like reduced toxicity risks while retaining targeted efficacy. They combine the innate cytotoxicity of natural killer (NK) cells with the targeted action of chimeric antigen receptors (CAR). Their potential lies in efficacy, tolerability and the possibility...

Introduction Indolent T‐cell lymphoma of the gastrointestinal tract (ITCL‐GI) is a rare primary T‐cell lymphoma from the gastrointestinal (GI) tract, there has been no documented successful treatment at the present time. Results A CD4(+)/CD8(−) ITCL‐GI with STAT3::JAK2 fusion was treated with Jakafi (Ruxolitinib), which resulted in significant cli...

Drug Discovery and Antiaging Approaches for Human Longevity explores antiaging biology and technical advancements in drug development for extending the human lifespan. It discusses the progress of clinical trials and ethical and social issues in antiaging medicines. This book covers topics such as genetics, human lifespan, and natural medicine, and...

Missing data are inevitable in clinical trials, and trials that produce categorical ordinal responses are not exempted from this. Typically, missing values in the data occur due to different missing mechanisms, such as missing completely at random, missing at random, and missing not at random. Under a specific missing data regime, when the conditio...

Automated insulin delivery (AID) systems-which integrate an insulin pump, continuous glucose monitoring (CGM), and a sophisticated algorithm to automate insulin delivery-have revolutionized the management of type 1 diabetes (T1D). AID systems aim to improve gly-caemic control and reduce the burden of manual management, and numerous clinical trials...

Conventional methods for analyzing composite endpoints in clinical trials often only focus on the time to the first occurrence of all events in the composite. Therefore, they have inherent limitations because the individual patients’ first event can be the outcome of lesser clinical importance. To overcome this limitation, the concept of the win ra...

Most adults with Down syndrome (DS) will develop pathology associated with Alzheimer's disease (AD). By age 65, the cumulative incidence of dementia exceeds 90%. The available data from trials in typically developed adults suggest the potential for intentional weight loss and changes in dietary intake to prevent or delay the development of AD. Howe...

The conventional approach to developing medical treatments and medical devices usually covers pre-clinical and in-vitro investigations, in-vivo animal studies and clinical trials with humans. In-silico trials and virtual cohorts present a promising avenue for addressing the challenges inherent in clinical research and improving its efficiency. Desp...

Background Real‐world evidence for hypomethylating agent (HMA) + venetoclax 50 mg (VEN50) with voriconazole and posaconazole in acute myeloid leukemia (AML), is limited. Methods We evaluated outcomes of patients with newly‐diagnosed AML treated with HMA + VEN50 with either posaconazole ( n = 23) or voriconazole ( n = 95). Results We report that t...

Human papillomavirus (HPV), as the main virus causing these cancers, accounts for 5.2% of the global cancer rate. Many preventive vaccines against HPV have already appeared on the market, and nowadays more and more researchers are carrying out more studies on therapeutic vaccines against HPV. The purpose of this paper is to discuss the latest resea...

Background Polymelia, or accessory limb, is a rare congenital abnormality with unknown etiology. Here we report the case of an infant with an accessory limb, review the diagnostic and therapeutic consideration of such cases, and highlight the potential challenges in management of an accessory limb. Case presentation We report the case of a 2-month...

Dose optimization in oncology clinical trials has shifted from seeking the maximum tolerated dose to identifying the Optimal Biological Dose (OBD) that balances therapeutic benefits and risks across multiple clinical attributes. Existing advanced dose-finding methods can integrate multiple endpoints and compare dose levels but are often complex or...

There is a growing recognition of the need for clinical trials to safely and effectively deploy artificial intelligence (AI) in clinical settings. We introduce dynamic deployment as a framework for AI clinical trials tailored for the dynamic nature of large language models, making possible complex medical AI systems which continuously learn and ada...

The purpose of writing this article is to compare legal and medical sciences by combining them with further systematization and use. In today’s turbulent environment, the implementation of informatisation of the medical community, including their patients, remains a relevant research vector. Practical implementation of the results of civil society...

In this issue of Cancer, Cecchini et al. report findings from a phase 2, open‐label trial of olaparib in 30 patients with advanced cholangiocarcinoma with isocitrate dehydrogenase 1 and 2 mutations. Although the trial did not meet its primary end point, a subset of patients experienced prolonged clinical benefit, which highlights the potential for...

Zusammenfassung Bakteriophagen stellen aufgrund ihrer biologischen Eigenschaften regulatorisch eine Besonderheit und gleichzeitig eine Herausforderung für die Arzneimittelzulassung dar. Etablierte europäische Leitlinien zur pharmazeutischen Qualität, Präklinik und Klinik greifen nur bedingt. Die steigende Bedrohung durch Infektionen mit multiresist...

We commend Jianwen Guo and colleagues’ study reporting the clinical trial results of the traditional Chinese medicine (TCM) compound FYTF-919 in the treatment of acute intracerebral haemorrhage. However, there are aspects of the study design that warrant further discussion.

Summarising the outcomes of the ESOLED trial investigating the efficacy and safety of olive leaf extract (Olea europaea) for glycemic control in patients with Type 2 diabetes.

Osteoarthritis (OA) is a degenerative joint disease that significantly impacts quality of life and poses a growing economic burden. Adipose tissue-derived therapies, including both cell-based and cell-free products, have shown promising potential in promoting cartilage repair, modulating inflammation, and improving joint function. Recent studies an...

Clinical trials of three poly (ADP‐ribose) polymerase (PARP) inhibitors, olaparib, niraparib and fuzuloparib, in platinum‐sensitive recurrent ovarian cancer (PSR OC) in China showed that PARP inhibitors improved progression‐free survival and achieved an all‐comer indication in this population. We reviewed the efficacy and safety of these PARP inhib...

The interplay of recipient age and graft loss causes is underexplored, despite its relevance for patient management and endpoint definition in clinical trials. This study aimed to investigate the impact of recipient age on graft loss causes. In this retrospective single-center cohort study with 1743 kidney transplantations between 1995 and 2022, gr...

Breaking up periods of sedentary time with brief bouts of high‐intensity interval exercise (HIIE) is suggested as a time‐efficient approach to improve exercise adherence and health. This randomized crossover clinical trial was designed to compare energy expenditure (EE), cardiometabolic, and perceptual responses during HIIE performed in a single se...

Small-conductance (K Ca 2.2) and intermediate-conductance (K Ca 3.1) Ca ²⁺ -activated K ⁺ channels are gated by a Ca ²⁺ -calmodulin dependent mechanism. NS309 potentiates the activity of both K Ca 2.2 and K Ca 3.1, while rimtuzalcap selectively activates K Ca 2.2. Rimtuzalcap has been used in clinical trials for the treatment of spinocerebellar ata...

Natural Language Processing (NLP) enables biopharma firms to mine unstructured data for competitive intelligence. This study explores how NLP techniques can analyze scientific literature, patents, clinical trial registries, and news to uncover trends, threats, and opportunities. The paper proposes an NLP framework to enhance strategic planning and...

To deliver high-quality goods and services that fully satisfy the needs and expectations of their clients, clinical trial sponsors and contract research organizations must establish, maintain, and closely monitor their QC and QA systems, as well as their integral standard operating procedures and other quality documents. The primary quality systems...

Chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) are recognized as a single disease entity with distinct phenotypic manifestations, characterized by predominant peripheral blood (PB) involvement in CLL and nodal and/or splenic disease in SLL1-3. This article explores the historically divergent approaches taken to treat CLL an...

Purpose This study investigated the accessibility of 3D reconstruction in the fixed coordinate system(3D-R-FCS) based on the basal anterior cortex of the femoral neck in measuring the femoral neck anteversion angle (FNAA). Methods CT and EOS examinations were performed on 40 femoral samples synthesized by Sawbone. Two evaluators were responsible f...

In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing phase I clinical trial of ALN-HTT02 from Alynlam Pharmaceuticals. We also report on the SAGE-718 (also known as dalzanemdor) program from Sage Therapeutics, with results of the phase II DIMENSION study and the recent termination of the open-label phase III...

Purpose The improved prognosis of rectal cancer through modern therapeutic approaches raises questions regarding quality of life (QoL) and functional outcomes. In this study, we compared post-transanal total mesorectal excision (taTME) short- and long-term QoL and functional outcomes with those after abdominal TME (abTME). Methods Prospective data...

Over the past three decades the quest for the testing of new chemical entities for emerging and old diseases without therapeutic drugs has increased. The uses of randomized controlled trials (RCTs) have become more popular over clinical judgment, case reports, and observational studies and became the gold evidential standard in medicine. In additio...

Background This secondary analysis of the ARAMIS trial evaluated renal function’s modifying effects on therapeutic responses to dual antiplatelet therapy (DAPT) versus intravenous thrombolysis in acute minor ischemic stroke. Methods Based on the as-treated set, we stratified patients by admission estimated glomerular filtration rate into three gro...

While clinical trials have shown the efficacy of using dupilumab and upadacitinib to treat atopic dermatitis (AD), long-term real-world data remain limited. This retrospective cohort study, utilizing data from the Polish National Health Fund Registry, evaluates the long-term effectiveness of dupilumab versus upadacitinib in early treatment responde...

The objective of this study was to compare the efficacy of a shorter (8 sessions) and longer (16 sessions) Dog-Assisted Therapy (DAT) program for children and adolescents with Fetal Alcohol Spectrum Disorder (FASD). We evaluated the impact of DAT on social skills, internalizing and externalizing problems, quality of life, severity of the disorder,...

Background and Objectives: Peripheral nerve stimulation (PNS) demonstrates promise for some neurological conditions. However, its effectiveness for essential tremor requires further research. In this randomized, double-blind, sham-controlled trial, we investigated the safety and efficacy of a single, 40-minute PNS session in individuals with essent...

Background Treatment with fluocinolone acetonide, hydroquinone, and tretinoin cream is the gold standard for melasma; however, the effects of this treatment in the Chinese population remain unclear. Due to the differences between Chinese and Caucasian subjects, further clinical trials in Chinese patients with melasma are needed. Aim To evaluate th...

Zusammenfassung Die Bakteriophagentherapie (kurz: Phagentherapie) zeigt großes Potenzial, den Problemen der uns bedrohenden zunehmenden Infektionen mit Multiantibiotikaresistenzen entgegenzuwirken. Registerstudien und systematische Übersichtsarbeiten zeigen, dass Phagenbehandlungen für diverse Indikationen bei etwa 80–90 % der antibiotikaresistente...

A high‐quality protocol document is essential for the successful and efficient implementation of clinical trials, but there is no consensus on how clinical trial protocol document quality should be evaluated. We used a modified Delphi approach and cognitive interviews to develop a new protocol document quality assessment tool, the Protocol Quality...

Objective While PD-L1 expression serves as a predictive biomarker for programmed cell death 1 and its ligand (PD-1/PD-L1) inhibitor efficacy in patients with non-small cell lung cancer (NSCLC), its association with treatment-related adverse events (TRAEs) has yet to be fully elucidated. This study systematically evaluated the correlation between PD...

La prise en charge des hémopathies malignes durant la grossesse représente un défi médical complexe. Ces maladies rares nécessitent une collaboration multidisciplinaire pour gérer à la fois les risques maternels liés aux changements physiologiques et les risques fœtaux, tels que les malformations ou la prématurité. Les données pour guider la prise...

Differences in the efficacy and safety of tyrosine kinase inhibitors (TKIs) have been observed across ethnic/ancestry subpopulations (previously reviewed to 2017). With an expanding number of TKIs approved since that time, an updated review of TKI response across ethnic/ancestry subpopulations in Phase 3 TKI clinical trials was conducted. A total o...

Ultra-high dose rate flash radiotherapy (FLASH-RT) has attracted wide attention in the field of radiotherapy in recent years. For FLASH-RT, radiation is delivered at a very high dose rate [usually thousands of times compared with conventional radiotherapy (CONV-RT)] in an extremely short time. This novel irradiation technique shows a protective eff...

The power to interpret the results of randomised clinical trial results relies on transparent reporting of the study design, protocol, methods and analyses. Without such clarity, the benefits of the findings, to both healthcare, policy and research, cannot be realized in full. The publication of the updated CONSORT 2025 and SPIRIT 2025 statements f...

Background: Cognitive impairment is a core feature of schizophrenia, profoundly impacting patients’ functional abilities. As such, evaluating cognition-related functional activity/impairment is essential for identifying effective treatments. This study presents findings from a non-interventional quantitative study to assess the inter-rater reliabil...

Background Low physical activity (LPA) is a major contributor to the global burden of chronic kidney disease (CKD). Our goal was to assess the spatiotemporal trends in the CKD burden attributable to LPA from 1990 to 2021, with a focus on the globe, China, five SDI regions, and four continents. Methods We analysed CKD-related deaths, DALYs, the ASM...

Wearable technologies and remote monitoring systems are transforming oncology by enabling continuous patient assessment, improving personalized care, and enhancing clinical trial efficiency. These innovations hold the potential to revolutionize cancer care delivery by providing real-time insights into patient health and treatment responses (Khan, 2...

Background Although evidence-based practices are crucial for enhancing nursing abilities and patient outcomes, many nurses encounter barriers and facilitators that limit their capacity to effectively apply evidence-based practices. Self-efficacy is crucial to how nurses view and overcome these challenges. Aim This study aimed to examine the relatio...