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Clinical Trials - Science topic

Clinical Trials, Bioequivalence and Bioavailability Studies, Clinical Studies
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The benefit from immune checkpoint inhibitors is tempered by immunologic toxicities, which involve diverse organs, have varying biology, onset time, and severity. Herein, we identify important areas of controversy and open research questions in the field of immune-related toxicity.
Article
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Polygenic scores can identify individuals with high disease risk based on inborn DNA variation. We explore their potential to enrich clinical trials by identifying individuals based on higher risk of disease (‘prognostic enrichment’), or increased probability of benefit (‘predictive enrichment’).
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An increasing prevalence of data-sharing models, aimed at making individual participant data (IPD) from clinical trials widely available, should facilitate the conduct of systematic reviews and meta-analyses based on IPD. We have assessed these different data-sharing approaches, from the perspective of experienced IPD reviewers, to examine their ut...
Article
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Background Dyslexia is characterized by difficulties in spelling and reading. The aim of this study is to identify domains of cognitive strength and weakness of students with dyslexia and to study the effect of an especially designed computerized training program on their reading abilities. This study was conducted on 2nd-grade primary school stude...
Article
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Orphan drug development is a rapidly expanding field. Nevertheless, clinical trials for rare diseases can present inherent challenges. Optimal study design and partnerships between academia and industry are therefore required for the successful development, delivery and clinical approval of effective therapies in this group of disorders.
Article
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Background Eptinezumab 100 mg and 300 mg met the primary efficacy endpoint in both PROMISE clinical trials, significantly reducing frequency of monthly migraine days over Weeks 1‒12. The objective of this analysis was to assess the clinical response to eptinezumab 100 mg and 300 mg within the pivotal phase 3 PROMISE-1 and PROMISE-2 studies to poten...
Article
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Pancreatic β cells differentiated from stem cells provide promise for cell replacement therapy of diabetes. Human pluripotent stem cells could be differentiated into definitive endoderm, followed by pancreatic progenitors, and then subjected to endocrinal differentiation and maturation in a stepwise fashion. Many achievements have been made in maki...
Article
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A number of chimeric antigen receptor (CAR) T cell-based therapies are now approved by the FDA for the treatment of cancer. A study published in Nature found that CAR T cells are still present in two patients who remain cancer-free over a decade after they received CAR T cell therapy as part of a clinical trial.
Article
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Analysis of multiple secondary outcomes in a clinical trial leads to an increased probability of at least one false significant result among all secondary outcomes studied. In this paper, we question the notion that that if no multiplicity adjustment has been applied to multiple secondary outcome analyses in a clinical trial, then they must necessa...
Article
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Background The benefits of preventive treatment on the effectiveness of migraine management have rarely been examined. This post hoc analysis investigated the impact of eptinezumab on the optimization of acute medication effectiveness using the 4-item Migraine Treatment Optimization Questionnaire (mTOQ-4) to measure acute medication optimization ov...
Article
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There is considerable variability in disease progression for patients with amyotrophic lateral sclerosis (ALS) including the age of disease onset, site of disease onset, and survival time. There is growing evidence that short structural variations (SSVs) residing in frequently overlooked genomic regions can contribute to complex disease mechanisms...
Article
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The selection of appropriate outcome measures is fundamental to the design of any successful clinical trial. Although dementia with Lewy bodies (DLB) is one of the most common neurodegenerative conditions, assessment of therapeutic benefit in clinical trials often relies on tools developed for other conditions, such as Alzheimer’s or Parkinson’s di...
Article
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Background Helicobacter pylori (H. pylori) infects ~ 35% of Americans and can lead to serious sequelae if left untreated. Growing evidence indicates that clarithromycin-based therapies (CBT) are becoming increasingly ineffective for treating H. pylori infection. RHB-105 was approved by the US Food and Drug Administration in 2019 for the treatment o...
Article
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This editorial describes the clinical trials related to antidiabetic drugs, most of them following an “add-on” design of where the new drug is added to metformin and the comparative arm is metformin plus placebo. Many drugs are already approved for therapy following this design; the authors believe that it is unethical to continue this trend becaus...
Article
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Chronic pain trials commonly allow auxiliary pain medications such as rescue and concomitant analgesics in addition to the randomized treatment. Changes in auxiliary pain medications after randomization represent intercurrent events that may affect either the interpretation or the existence of the measurements associated with the clinical question...
Article
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Hepatitis B virus (HBV) infected about 296 million people worldwidely, while clinical useful therapeutic agents were still limited. Capsid assembly modulators (CAM) have been validated as efficient anti-HBV virus agents with the potential to achieve functional cure, therefore piqued much attention in recent years. There are various novel scaffold a...
Article
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Purpose of Review In this review, we have focused on the currently available transcatheter tricuspid valve therapies, device selection, as well as role and management of tricuspid regurgitation (TR) in the setting of other transcatheter valvular procedures. Recent Findings In this review, we have enlisted the recently finished as well as ongoing t...
Article
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In this article, we review the fundamentals and current clinical data regarding suprachoroidal corticosteroid injections for the management of macular edema. Recently conducted clinical trials have demonstrated both the efficacy, duration, and favorable side effect profile of suprachoroidal injections in managing macular edema due to varying ocular...
Article
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Due to concerns about the safety and efficacy of long-term opioid treatment, non-pharmacological interventions for chronic pain such as cognitive-behavioral therapy for chronic pain (CBT-CP) are being promoted as first-line treatments. Telehealth delivery is designed to increase access and reduce barriers to CBT-CP with comparable effectiveness rel...
Research
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To Study the efficacy of Panchanimba Choorna & ArkaTaila by conducting clinical trials on Vicharchika W.S.R. to Eczema & To see side effects or adverse effects (if any) of Panchanimba Choorna & ArkaTaila during clinical trials. Results in Comparing all the symptoms before and after treatment of three groups had significant action in management of V...
Article
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El objetivo de este trabajo de investigación fue determinar la aplicación de la terapia celular en la Esclerosis Múltiple. A lo largo del proceso de investigación se recopiló información especializada y de fuentes verificadas. De esta manera se encontró que en estos últimos años se han realizado diversos ensayos clínicos con trasplantes de células...
Research
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To Study the efficacy of Sammoha Loha, Navayas Loha, and Mustadi Churna, by conducting clinical trials on Pandu Vyadhi W.S.R. to Anaemia& To see side effects or adverse effects (if any) of Sammoha Loha, Navayas Loha, and Mustadi Churna during clinical trials. Results in comparing all the symptoms before and after treatment of three groups had signi...
Article
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Recurrent events refer to events that over time can occur several times for each individual. Full use of such data in a clinical trial requires a method that addresses the dependence between events. For modelling this dependence, there are two time scales to consider, namely time since start of the study (running time) or time since most recent eve...
Article
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Ivosidenib, an inhibitor of isocitrate dehydrogenase 1 (IDH1) R132C and R132H variants, is approved for the treatment of acute myeloid leukaemia (AML). Resistance to ivosidenib due to a second site mutation of IDH1 R132C, leading to IDH1 R132C/S280F, has emerged. We describe biochemical, crystallographic, and cellular studies on the IDH1 R132C/S280...
Article
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Understanding the loss of kidney function resulting from kidney aging has become an emerging research focus that will facilitate the future development of antisenolytic treatments. In this issue of the JCI, Pippin et al. first identified PD-1 upregulation in the aged mouse podocyte via unbiased RNA-seq analysis. Overexpression of PD-1 in immortaliz...
Article
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Regulatory T cells (Tregs) have enormous therapeutic potential to treat a variety of immunopathologies characterised by aberrant immune activation. Adoptive transfer of ex vivo expanded autologous Tregs continues to progress through mid- to late-phase clinical trials in several disease spaces and has generated promising preliminary safety and effic...
Article
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Sympathetic overactivation is one of the main contributors to development and progress of hypertension. Renal denervation (RDN) has been evidenced by series of clinical trials for its efficacy and safety to treat overactivated sympathetic nervous system induced diseases. However, the results were inconsistent and not all patients benefited from RDN...
Article
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Myelofibrosis (MF) is a clonal myeloproliferative neoplasm, typically associated with disease-related symptoms, splenomegaly, cytopenias and bone marrow fibrosis. Patients experience a significant symptom burden and a reduced life expectancy. Patients with MF receive ruxolitinib as the current standard of care, but the depth and durability of respo...
Article
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Azacitidine and decitabine are hypomethylating agents that have dose‐dependent epigenetic and cytotoxic effects and are widely used in the treatment of myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). In this review, we discuss the path to regulatory approval of azacitidine and decitabine, highlighting the substantial efforts that...
Article
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Adaptive designs are a class of methods for improving efficiency and patient benefit of clinical trials. Although their use has increased in recent years, research suggests they are not used in many situations where they have potential to bring benefit. One barrier to their more widespread use is a lack of understanding about how the choice to use...
Presentation
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In this episode of Hematopoiesis, Dr. Alexis Caulier has exciting conversations with three experts in the field about how early bench work on the genetic regulation of fetal hemoglobin led to the development of bedside gene therapies. Dr. David Nathan revisits the beneficial role of fetal hemoglobin to improve the clinical manifestations of hemoglo...
Research Proposal
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Significant changes in relation to an increased "nutritional awareness" of the consumer have recently been observed. The role of food producers is no longer limited to providing consumers with a wide range of products, but their composition is also not without significance. There is an increase in interest in so-called "functional food", which, in...
Article
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Coronavirus disease 2019 (COVID-19) has rapidly spread around the world since December 2019, becoming a global pandemic. Atypical cases of COVID-19, manifesting as prolonged positive SARS-CoV-2 test results during the convalescence period, have been encountered. These cases increase the difficulty of COVID-19 prevention and treatment. Here, we repo...
Article
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There is a growing interest in precision medicine, where a potentially censored survival time is often the most important outcome of interest. To discover optimal treatment regimens for such an outcome, we propose a semiparametric proportional hazards model by incorporating the interaction between treatment and a single index of covariates through...
Chapter
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This chapter presents the work carried out to determine the suitability and effectiveness of the new compression wrap for the treatment of the lower limb venous disease and lympoedema. First, it describes the clinical trials used to test the clinical effectiveness and tolerability of the new compression wrap. Secondly, it describes the survey condu...
Article
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So regularly, Continues and untimely consuming Viruddhahar for long time there is formation of Garavisha results in Aganimandya leading to dosh dusti especially Pitta Dosha and dhatu dusti leading to dhatushaithilya resulting Bal, Varna and Ojakashya and subsequently manifests as Pandu showing symptoms as Daurbalya, Annadavit, Shram, Bhram, Aruchi,...
Article
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Vatarakta is caused due to avarana pathology in Vatta dosha and Rakta Dhatu.In conventional medicine, Vataraktaresembles with Gout.In this study, 30 clinically diagnosedpatients of vatarakta were selected. 20 ml of Guduchi Ghrita wasadministered orally twice a day before meal with warm water.Follow-up was taken weekly till 28 th days.The statistica...
Article
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Dietary pattern of Indian people has been changing upto certain extent by the influence of western culture. Irregular and untimely eating habits along with certain variations of recipes can be included under the Viruddhaahar. Vagbhatacharya quotes that the effects of Viruddhahar consumption are likely that of Garavish. Amlapitta is one of the disea...
Article
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Background: Guselkumab is an IL-23 inhibitor that has demonstrated to be effective and safe for the treatment of moderate-to-severe plaque psoriasis in clinical trials. The data pool relating to the use of Guselkumab in a real-life setting is still lacking. Objective: To evaluate efficacy and safety of Guselkumab in a real-life setting, focusing...
Article
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VranaShopha is one of the commonest clinical conditions, which we come across in our day-today surgical practice. Sthanik Shopha is also termed as VranaShopha. This clinical entity is characterized by Vedana, Utseda, Sthanik Ushma Vrudhi and Vivarnata. The disease has three different stages as Ama, Pachyamana and Pakwa avastha. The sufferer experie...
Article
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Here we describe the development and evaluation of a survey instrument to assess the research suitability of veterinary electronic medical records (EMRs) through the conduct of two studies as part of the Dog Aging Project (DAP). In study 1, four reviewers used the instrument to score a total of 218 records in an overlapping matrix of pairs to asses...
Article
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Purpose of Review Despite being uncommon, breast cancer at a young age correlates with worse outcomes. Several aspects of a breast cancer diagnosis may affect young and older patients differently, including biology, genetic predisposition, fertility concerns, and psychosocial outcomes. Understanding these differences is critical to providing tailor...
Preprint
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AI-assisted colonoscopy has received lots of attention in the last decade. Several randomised clinical trials in the previous two years showed exciting results of the improving detection rate of polyps. However, current commercial AI-assisted colonoscopy systems focus on providing visual assistance for detecting polyps during colonoscopy. There is...
Article
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Long COVID-19 has been defined as the condition occurring in individuals with a history of probable or confirmed SARS-CoV-2 infection, with related symptoms lasting at least 2 months and not explainable by an alternative diagnosis. The practice of digital physiotherapy presents itself as a promising complementary treatment method to standard physio...
Article
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Clinical trials generate key evidence to inform decision making, and also benefit participants directly. However, clinical trials frequently fail, often struggle to enroll participants, and are expensive. Part of the problem with trial conduct may be the disconnected nature of clinical trials, preventing rapid data sharing, generation of insights a...
Article
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It is well established from clinical trials that behavioural interventions can halve the risk of progression from prediabetes to type 2 diabetes but translating this evidence of efficacy into effective real-world interventions at scale is an ongoing challenge. A common suggestion is that future preventive interventions need to be more personalised...
Article
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Measurement of upper limb function is critical for tracking clinical severity in amyotrophic lateral sclerosis (ALS). The Amyotrophic Lateral Sclerosis Rating Scale-revised (ALSFRS-r) is the primary outcome measure utilised in clinical trials and research in ALS. This scale is limited by floor and ceiling effects within subscales, such that clinica...
Article
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Human pathophysiology is occasionally too complex for unaided hypothetical-deductive reasoning and the isolated application of additive or linear statistical methods. Clustering algorithms use input data patterns and distributions to form groups of similar patients or diseases that share distinct properties. Although clinicians frequently perform t...
Article
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Pain is one of the most common symptoms in cancer patients including older adults. The objective of this study was to evaluate the enrollment criteria that can limit the inclusion of older adults in clinical trials concerning cancer-related pain (CRP). The study included 356 trials registered with ClinicalTrials.gov. Our primary outcome measures we...
Article
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HIV incidence among women in Eastern and Southern Africa remains unacceptably high, highlighting the need for effective HIV prevention options, including pre-exposure prophylaxis (PrEP). The Evidence for Contraceptive Options and HIV Outcomes trial offered daily oral PrEP to participants during the latter part of the clinical trial as an additional...
Article
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Clinical trials for Alzheimer's disease (AD) are slower to enroll study participants, take longer to complete, and are more expensive than trials in most other therapeutic areas. The recruitment and retention of a large number of qualified, diverse volunteers to participate in clinical research studies remain among the key barriers to the successfu...
Article
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Cell-free protein synthesis (CFPS) technologies have grown from lab-scale research tools to biopharmaceutical production at the Good Manufacturing Practice manufacturing scale. Multiple human clinical trials are in progress with CFPS-based products. In addition, applications of CFPS in research have continued to expand over the years and play an im...
Article
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Introduction Supratotal resection (SpTR) of glioblastoma may be associated with improved survival, but published results have varied in part from lack of consensus on the definition and appropriate use of SpTR. A previous small survey of neurosurgical oncologists with expertise performing SpTR found resection 1–2 cm beyond contrast enhancement was...
Article
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The repeated occurrence of the same event in a process is commonly observed in many domains. Such events are referred to as recurrent events. The time to occurrence of these repeated events varies from unit to unit with a possibility of events not occurring among some of the units. Invariably such data are dealt with using some of the techniques in...
Article
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Introduction: Supplementary feeding programme is a strategy for managing under-fives with moderate acute malnutrition (MAM). This study aimed to determine the effect of adherence to follow-up on recovery from MAM among under-fives. Methods: A clinical trial to evaluate the effectiveness of daily supplementary rations of a standardised milk-based fo...
Preprint
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The primary analysis of clinical trials in diabetes therapeutic area often involves a mixed-model repeated measure (MMRM) approach to estimate the average treatment effect for longitudinal continuous outcome, and a generalized linear mixed model (GLMM) approach for longitudinal binary outcome. In this paper, we considered another estimator of the a...
Article
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Eligibility criteria for clinical trials are intended to select suitable study subjects but can limit trial participation and generalization of results. While reported for other cancers, non-enrollment rates and evolution of eligibility criteria over time have so far not been studied for randomized controlled trials (RCTs) involving adults with acu...
Preprint
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Aim We evaluated pembrolizumab monotherapy in patients with advanced salivary gland carcinoma on the phase 2 KEYNOTE-158 study (NCT02628067)