Journal of clinical trials最新文献

{"title":"A Double-Blind Randomized, Placebo-Controlled Phase 2 Clinical Trial to Evaluate Safety and Efficacy of US APR 2020 in Subjects with ChronicKidney Disease Stage IV","authors":"Emmanuel A. Anteyi, P. Ranganathan, U. Vyas, Qianchuan Zhao, N. Ranganathan","doi":"10.35248/2167-0870.21.11.468","DOIUrl":"https://doi.org/10.35248/2167-0870.21.11.468","url":null,"abstract":"Background: CKD is a common public health problem affecting over 200 million people worldwide. The United States accounted for about 30 million CKD patients with high morbidity, mortality and enormous health costs. Despite advancement in treatment options of risk factors for CKD like hypertension and diabetes, progression to end stage kidney disease and complications remain high. Observations of gut dysbiosis associated with progressive kidney disease, systemic inflammation and uremic toxin retention led to trial use of probiotics in restoring gut microbiome to ameliorate CKD. Studies have shown benefits in restoring gut microbiota with probiotics retarded progression, reduced uremic toxins, and improved quality of life in CKD. This clinical trial is to evaluate safety and efficacy of probiotic formulation US APR 2020 in patients with CKD stage 4 with an aim of retarding progression of CKD to End Stage Kidney Disease (ESRD). Methods: All eligible adult patients with CKD (eGFR 15-29 mls/min) with serum creatinine >2.5 mg/dl will be randomly assigned to either US APR 2020 (Group A) or Placebo (group B). The participants will be followed up monthly from baseline to 6 months of end of treatment with schedule of assessments at each visit. The primary end points will be based on <10% of adverse events and mean reduction of 40% decline in eGFR between the two groups at end of treatment. Discussion: This trial protocol outlined the design to evaluate Phase 2 safety and efficacy of FDA approved probiotic IND for treatment of patients in CKD stage 4 with primary objective of retarding renal disease progression. Data from this trial will inform planning for a Phase 3 clinical trial as part of unique and innovative drug development program for these categories of patients.","PeriodicalId":15375,"journal":{"name":"Journal of clinical trials","volume":"3 1","pages":"1-7"},"PeriodicalIF":0.0,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"76217623","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical Trials in Covid-19 Disease","authors":"Abigail Mk","doi":"10.35248/2167-0870.21.S9.E005","DOIUrl":"https://doi.org/10.35248/2167-0870.21.S9.E005","url":null,"abstract":"","PeriodicalId":15375,"journal":{"name":"Journal of clinical trials","volume":"3 1","pages":"1-1"},"PeriodicalIF":0.0,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"84092563","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Investigating Synthetic Controls with Randomized Clinical Trial Data in Rheumatoid Arthritis Studies","authors":"Zailong Wang, Zhuqing Yu, Su Chen, Lanju Zhang","doi":"10.35248/2167-0870.21.11.466","DOIUrl":"https://doi.org/10.35248/2167-0870.21.11.466","url":null,"abstract":"The cost of clinical research for new drug development has been increasing rapidly. An effective approach to reduce the cost of clinical trials is to use a synthetic control arm to substitute a concurrent control arm. Synthetic control arms are usually created with propensity-score-based methods from historical or external patient-level control data. Although there is much literature discussing how to create synthetic control arms, little is known about how synthetic control arms perform compared to concurrent control arms in real clinical trials. In this paper, we take a real randomized controlled clinical trial and create a synthetic control arm for it using propensity-score-based methods from the control data in other randomized clinical trials. The goal is to demonstrate validity of using synthetic control arms by comparing the performance of synthetic control arms to the concurrent control arm. Four propensity-score-based methods, stratification, matching, inverse probability of treatment weighting, and covariate adjustment are applied to create the synthetic control group. Our results show that the synthetic control arm created with the stratification or matching method could provide an estimate of treatment effect that is as accurate as that of a real randomized clinical trial. This suggests a good opportunity to expedite drug development with reduced cost. We encourage use of these methods in clinical research for drug development when patient-level control data from comparable historical randomized clinical trials are available.","PeriodicalId":15375,"journal":{"name":"Journal of clinical trials","volume":"23 1","pages":"1-9"},"PeriodicalIF":0.0,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"89665776","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effects of COVID-19 on Mental Health","authors":"S. Won","doi":"10.35248/2167-0870.21.S11.E001","DOIUrl":"https://doi.org/10.35248/2167-0870.21.S11.E001","url":null,"abstract":"","PeriodicalId":15375,"journal":{"name":"Journal of clinical trials","volume":"5 1","pages":"1-1"},"PeriodicalIF":0.0,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"85109407","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment of COVID-19 Patients at Intensive Care Unit of General Hospital of Prizren","authors":"Afrim Avdaj, Anisa Mukaj, A. Bytyqi, Artur Avdaj, Syla Osmanaj, Mentor Rexhbeqaj, Anila Cake, Agron Bytyqi","doi":"10.35248/2167-0870.21.11.463","DOIUrl":"https://doi.org/10.35248/2167-0870.21.11.463","url":null,"abstract":"Introduction: Due to the COVID-19 pandemic, hospitals and especially the intensive care unit are and can be overloaded by the hypoxemic patients, and face many challenges. Methods: There was used retrospective observational study in a Kosovo hospital, the review of characteristics, clinical course and outcomes of all consecutive patients who had respiratory failure. In addition to data collected in ICU of patients with worsening symptoms and COVID-19 confirmed, similar publications in medical journals with keywords, coronavirus, SARS-CoV2, intensive care and treatment have also been reviewed. Results: During July, August and September, 797 confirmed patients with COVID-19 were admitted to the hospital, of which ninety-four patients (11.79%) were treated in the Central ICU of Prizren General Hospital. 59.58% were male, the youngest age was 34 years old, the oldest 84 and the average was 65.53 years old. Regarding the days of stay there were 0 up to 21 days of stay, the average was 5.06 days of stay. Out of 94 patients admitted 13 (13.83%) were discharged in improved condition at home, 19 (20.21%) were transferred out of ICU and 62 (65.96%) have died. The youngest of the dead was 46 years. while the oldest was 84 y, the average age of the dead was 68.06 years old. Conclusion: It is needed to adapt management and safe treatment protocols as well as the demand for multidisciplinary treatment. Patients with COVID-19 who need to be transferred to the ICU are complex and have a high mortality rate.","PeriodicalId":15375,"journal":{"name":"Journal of clinical trials","volume":"32 1","pages":"1-4"},"PeriodicalIF":0.0,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"88527196","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Coronavirus Disease 2019 and Androgens: An Intriguing Association","authors":"Aless, ra Renieri, M. Baldassarri, M. Carriero, A. Isidori, M. Marcelli","doi":"10.35248/2167-0870.21.S9.001","DOIUrl":"https://doi.org/10.35248/2167-0870.21.S9.001","url":null,"abstract":"","PeriodicalId":15375,"journal":{"name":"Journal of clinical trials","volume":"99 1","pages":"1-2"},"PeriodicalIF":0.0,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"75382325","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Non-Invasive Strategies for Nose-to-Brain Drug Delivery","authors":"Trevino Ja, Rodrigo C. Quispe, Faiza A. Khan, Novak","doi":"10.35248/2167-0870.10.7.439","DOIUrl":"https://doi.org/10.35248/2167-0870.10.7.439","url":null,"abstract":"Intranasal drug administration is a promising method for delivering drugs directly to the brain. Animal studies have described pathways and potential brain targets, but nose-to-brain delivery and treatment efficacy in humans remains debated. We describe the proposed pathways and barriers for nose-to-brain drug delivery in humans, drug properties that influence central nervous system delivery, clinically tested methods to enhance absorption, and the devices used in clinical trials. This review compiles the available evidence for nose-to-brain drug delivery in humans and summarizes the factors involved in nose-to-brain drug delivery.","PeriodicalId":15375,"journal":{"name":"Journal of clinical trials","volume":"1 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2020-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"83013484","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Early Sequential Risk Stratification Assessment to Optimize Fluid Dosing, CRRT Initiation and Discontinuation in Critically Ill Children with Acute Kidney Injury: Taking Focus 2 Process Article","authors":"Jean-Philippe Roy, K. Krallman, Rajit K. Basu, R. Chima, Lin Fei, Sarah Wilder, A. Schmerge, Bradley Gerhardt, Kaylee Fox, Cassie L. Kirby, S. Goldstein","doi":"10.35248/2167-0870.20.10.435","DOIUrl":"https://doi.org/10.35248/2167-0870.20.10.435","url":null,"abstract":"Background: Acute Kidney Injury (AKI) is common in critically ill children and is associated with increased morbidity and mortality. Recognition and management of AKI is often delayed, predisposing patients to risk of clinically significant fluid accumulation (Fluid Overload (FO)). Early recognition and intervention in high risk patients could decrease fluid associated morbidity. We aim to assess an AKI Clinical Decision Algorithm (CDA) using a sequential risk stratification strategy integrating the Renal Angina Index (RAI), urine Neutrophil Gelatinase-Associated Lipocalin (NGAL) and the Furosemide Stress Test (FST) to optimize AKI and FO prediction and management in critically ill children. Methods/Design: This single center prospective observational cohort study evaluates the AKI CDA in a Pediatric Intensive Care Unit (PICU). Every patient ≥ 3 months old has the risk score RAI calculated automatically at 12 hours of admission. Patients with a RAI ≥ 8 (fulfilling renal angina) have risk further stratified with a urine NGAL and, if positive (NGAL ≥ 150ng/mL), subsequently by their response to a standardized dose of furosemide (namely FST). RAI negative or NGAL negative patients are treated per usual care. FST-responders are managed conservatively, while non-responders receive fluid restrictive strategy and/or continuous renal replacement therapy (CRRT) at 10%-15% of FO. 2100 patients over 3 years will be evaluated to capture 210 patients with severe AKI (KDIGO Stage 2 or 3 AKI), 100 patients with >10% FO, and 50 requiring CRRT. Primary analyses: Standardizing a pediatric FST and assessing prediction accuracy of CDA for severe AKI, FO>10% and CRRT requirement in children. Secondary analyses in patients with AKI: Renal function return to baseline, RRT and mortality within 28 days. Discussion: This will be the first prospective evaluation of feasibility of AKI CDA, integrating individual prediction tools in one cohesive and comprehensive approach, and its prediction of FO>10% and AKI, as well as the first to standardize the FST in the pediatric population. This will increase knowledge on current AKI prediction tools and provide actionable insight for early interventions in critically ill children based on their level of risk.","PeriodicalId":15375,"journal":{"name":"Journal of clinical trials","volume":"23 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2020-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"79506208","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Commentary on Treating Impulsivity with Probiotics in Adults (PROBIA): Study Protocol of a Multicenter, Double-blind, Randomized, Placebo-Controlled Trial","authors":"G. Arteaga-Henríquez, J. Ramos-Quiroga","doi":"10.35248/2167-0870.20.10.406","DOIUrl":"https://doi.org/10.35248/2167-0870.20.10.406","url":null,"abstract":"Gara Arteaga-Henríquez1,2*, J Antoni Ramos-Quiroga1,2,3,4 1Department of Psychiatric Genetics Unit, Vall d’Hebron Research Institute (VHIR), Barcelona, Catalonia, Spain;2Department of Psychiatry, Hospital Universitari Vall d’Hebron, Barcelona, Catalonia, Spain;3Department of Network Research Centre on Mental Health (CIBERSAM), Barcelona, Catalonia, Spain;4Department of Psychiatry and Legal Medicine, Universitat Autònoma de Barcelona, Barcelona, Catalonia, Spain STUDY DESCRIPTION","PeriodicalId":15375,"journal":{"name":"Journal of clinical trials","volume":"158 1","pages":"1-2"},"PeriodicalIF":0.0,"publicationDate":"2020-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"73496967","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Should Thrombus Aspiration be routinely used in STEMI with TIMI 0 Flow","authors":"B. Budiono","doi":"10.35248/2167-0870.20.10.436","DOIUrl":"https://doi.org/10.35248/2167-0870.20.10.436","url":null,"abstract":"Patients with pre-procedural TIMI flow grade 0 had a higher incidence of myocardial blush grade ≤ 1 and no reflow and had greater myocardial damage as assessed by peak creatine kinase-MB fraction value compared with those with pre-procedural TIMI flow grade 2 to 3 [1]. Several explanations may account for the potential significance of pre-procedural TIMI flow grade on clinical outcomes in patients with STEMI undergoing primary PCI. Prolonged ischemia and late reperfusion can impair endothelial function and cause myocardial tissue edema and hemorrhage. It might explain why optimal epicardial recanalization, primary angioplasty for STEMI is still associated with suboptimal reperfusion in a relatively large proportion of patients, especially with late onset [2]. Therefore, rapid restoration of the infarct-related coronary artery has become a main goal in patients with ST-segment–elevation myocardial infarction (STEMI) [3,4]. Distal embolization of atherothrombotic material during primary percutaneous coronary intervention for ST-elevation myocardial infarction is an important cause of (partly) unsuccessful reperfusion [5]. Reducing the thrombus burden by using thrombus aspiration catheter is rational concept in primary PCI. A study showed that distal embolization was associated with a 5-fold increase in 5-year mortality [6]. However, the use of routine thrombus aspiration called into a question by data indicating not only a lack of efficacy but a risk of potentially deleterious complications [7].","PeriodicalId":15375,"journal":{"name":"Journal of clinical trials","volume":"28 1","pages":"1-2"},"PeriodicalIF":0.0,"publicationDate":"2020-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"73986680","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}