Open Access Journal of Clinical Trials最新文献

{"title":"Terminated Interventional Trials in the Clinical Trial Registry of India Database: An Analysis to Evaluate the Reasons for Termination","authors":"S. Dutta, Rimplejeet Kaur, M. Haque, P. Bhardwaj, D. Saxena, N. Rahman, H. Lugova, Dilshad Jahan, Salequl Islam, T. Chowdhury, J. Charan","doi":"10.2147/oajct.s285177","DOIUrl":"https://doi.org/10.2147/oajct.s285177","url":null,"abstract":"","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"53 1-2","pages":"61-70"},"PeriodicalIF":1.2,"publicationDate":"2020-12-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41290378","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":"The Use of Gabapentin for the Treatment of Alcohol and Tobacco Use Disorders: A Review","authors":"Gabriel C Quintero Garzola","doi":"10.2147/OAJCT.S257556","DOIUrl":"https://doi.org/10.2147/OAJCT.S257556","url":null,"abstract":"Background: Alcohol and tobacco use disorders are pervasive health problems without adequate treatment. Gabapentin has been used for different psychiatric disorders, including addiction. Methods: In this article, the use of gabapentin for the treatment of alcohol and tobacco use disorders is reviewed. Accordingly, a database search of PubMed was performed (January 1, 1983–August 31, 2017), using the search terms “gabapentin” AND “alcohol” OR “tobacco”. Animal and human studies written in English were included. A total of 426 records were identified, and 40 articles met eligibility criteria and were included in this review. The main reasons for exclusion were gabapentin was not the intervention of interest or the study population did not have alcohol/tobacco abuse issues. Additionally, 28 references were included in the Introduction, for a total of 68 references. The success of gabapentin for treating alcohol/tobacco use problems was determined by calculating the success rate across studies: the total number of investigations with favorable results was divided by the total numbers of investigations for each specific disorder (eg, alcohol or tobacco dependence, or other). Results and Discussion: The available evidence on gabapentin for alcohol use disorder suggests that this treatment successfully alleviates problems related to alcohol abuse, including withdrawal symptoms, dependence, and cravings. However, more research is needed to determine the effectiveness of gabapentin for preventing relapse. Further, more data are required before a conclusion regarding gabapentin’s effectiveness for treating tobacco dependence.","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"1 1","pages":""},"PeriodicalIF":1.2,"publicationDate":"2020-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43086063","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":"Taking Optogenetics into the Human Brain: Opportunities and Challenges in Clinical Trial Design.","authors":"Michael White, Michael Mackay, Roger Whittaker","doi":"10.2147/OAJCT.S259702","DOIUrl":"10.2147/OAJCT.S259702","url":null,"abstract":"<p><p>Optogenetics, the use of light to control the activity of suitably sensitized cells, has led to major advances in the field of basic neuroscience since it first emerged in 2005. Already, the technique has entered clinical trials for conditions such as Retinitis Pigmentosa. A major focus of interest is the use of optogenetics within the brain, where the ability to precisely control the activity of specific subsets of neurons could lead to novel treatments for a wide range of disorders from epilepsy to schizophrenia. However, since any therapy would require both the use of gene therapy techniques to introduce non-human proteins, and implantable electronic devices to provide optical stimulation, applying this technique in the brain presents a unique set of obstacles and challenges. This review looks at the reasons why researchers are exploring the use of optogenetics within the brain. It then explores the challenges facing scientists, engineers and clinicians wanting to take this technology from the lab into the first human brain, discussing different possibilities for a first-in-human clinical trial from a sponsor, patient and regulatory perspective.</p>","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"2020 ","pages":"33-41"},"PeriodicalIF":1.2,"publicationDate":"2020-08-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7611592/pdf/EMS133514.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39377087","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Effectiveness of Body Awareness Therapy in Stroke Survivors: A Systematic Review of Randomized Controlled Trials","authors":"Abayneh Alamer, Kefale Getie, Haimanot Melese, Habtamu Mazea","doi":"10.2147/oajct.s260476","DOIUrl":"https://doi.org/10.2147/oajct.s260476","url":null,"abstract":": Body awareness therapy has been hypothesized as an effective program for maintaining balance and improving gait function in patients with neurological diseases. Nevertheless, there is a dearth of recent studies in a systematic way to confirm its efficacy on stroke survivors. The objective of this review was to synthesize the current evidence on the effectiveness of body awareness therapy on balance and gait function in stroke survivors. A comprehensive search of PubMed, CINAHL, AMED, PEDro, EMBASE, REHABDATA Database, and Scopus was done to identify randomized controlled trials of body awareness therapy in individuals with stroke. The PEDro scale was used to evaluate the methodological quality of included trials. The primary outcome measures of this review were the 10-Meter Walk Test (10MWT) and Berg Balance Scale (BBS). Due to varying included trials, meta-analysis was not carried out. Nine randomized controlled trials with 332 patients were analyzed. Body awareness therapy exhibited a moderate confirmation to better balance and gait function of stroke survivors. Body awareness therapy could be a well-founded rehabilitative intervention and might support enhanced balance and gait function of individuals with stroke.","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":" ","pages":""},"PeriodicalIF":1.2,"publicationDate":"2020-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/oajct.s260476","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48298515","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":"Remdesivir is Effective for Moderately Severe Patients: A Re-Analysis of the First Double-Blind, Placebo-Controlled, Randomized Trial on Remdesivir for Treatment of Severe COVID-19 Patients Conducted in Wuhan City","authors":"W. Shih, Xin Shen, Peng Zhang, Tai Xie","doi":"10.2147/oajct.s262606","DOIUrl":"https://doi.org/10.2147/oajct.s262606","url":null,"abstract":"Introduction: The first clinical trial on remdesivir for treatment of severe COVID-19 conducted in China was terminated prematurely due to limited patient enrollment, which rendered the findings inconclusive. We re-analyzed the efficacy with a statistically more powerful and clinically meaningful method based on published data using the 6-point ordinal scale of patient’s disease severity. Methods: We defined response as patient’s point reached, either 2 (hospitalized, no require-ment for supplementary oxygen therapy) or 1 (discharged or met discharge criterion), and then analyzed with logistic regression with baseline score, day of assessment, treatment group, baseline by treatment interaction, and day by treatment interaction as covariates. The binary endpoint was supported by the recent FDA’s guidance on COVID-19. Results: Eighty-two percent (82%) of the patients were in the disease severity point=3 (hospitalized, required supplemental oxygen (but not NIV/HFNC)) – the moderately severe category. The response rate was 85% for remdesivir-treated patients with baseline disease point=3 versus 70% response rate for likewise placebo-treated patients on Day 28 (OR=2.38, P=0.0012). On Day 14, the response rate for these patients was 43% for remdesivir versus 33% for placebo (OR=1.53, P=0.0022). For patients with baseline disease point=4 (critically severe category), no similar comparisons were statistically significant. Conclusion and Discussion: The Chinese trial was not really under-powered as previously perceived or portrayed by many opinions. This result supports the preliminary findings of ACTT that remdesivir is effective for patients who were not critically severe. This result also suggests that remdesivir should be given to hospitalized COVID-19 patients as soon as possible. There is no race difference in the treatment effect.","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"12 1","pages":"15-21"},"PeriodicalIF":1.2,"publicationDate":"2020-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/oajct.s262606","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43654704","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":"MCLENA-1: A Phase II Clinical Trial for the Assessment of Safety, Tolerability, and Efficacy of Lenalidomide in Patients with Mild Cognitive Impairment Due to Alzheimer's Disease.","authors":"Boris Decourt, Jeffrey Wilson, Aaron Ritter, Christopher Dardis, Frank P DiFilippo, Xiaowei Zhuang, Dietmar Cordes, Garam Lee, Nadia D Fulkerson, Tessa St Rose, Katurah Hartley, Marwan N Sabbagh","doi":"10.2147/oajct.s221914","DOIUrl":"10.2147/oajct.s221914","url":null,"abstract":"<p><p>With the general population reaching higher ages, a surge in Alzheimer's disease (AD) incidence will happen in the coming decades, putting a heavy burden on families and healthcare systems Worldwide. This emphasizes the pressing need for AD therapeutic interventions. Accumulating evidence indicates that inflammation is prominent both in the blood and central nervous system of AD sufferers. These data suggest that systemic inflammation plays a crucial role in the cause and effects of AD neuropathology. Capitalizing on our experience from a previous clinical trial with thalidomide, we hypothesize that modulating inflammation via the pleiotropic immunomodulator lenalidomide may alter AD if administered during a proper time window in the course of the disease. Thus, in this Phase II, proof-of mechanism study, 30 amnestic mild cognitive impairment (aMCI) subjects will be treated with lenalidomide at 10 mg/day for 12 months on a 1:1 ratio, followed by a 6 months washout period. The primary objective of this study is to investigate the effect of lenalidomide on cognition, which is assessed at regular intervals. The secondary objective is to assess the safety and tolerability of lenalidomide in aMCI patients evaluated through adverse events, vital signs, clinical biochemistry, and physical and neurological examinations. Tertiary objectives are to analyze the effects of lenalidomide on brain amyloid loads (Florbetapir PET imaging) and neurodegeneration (volumetric MRI) by comparing pre- and post-dosing data. Finally, exploratory objectives will investigate whether blood inflammatory markers can serve as surrogate markers of therapeutic efficacy. Our study should determine whether lenalidomide is safe in AD subjects and whether it can alter the clinical progression of AD when administered before dementia onset. If effective, lenalidomide would become the first drug capable of delaying the trajectory of AD, which could lead the way to find additional, less toxic treatments in the near future.</p>","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"12 ","pages":"1-13"},"PeriodicalIF":1.4,"publicationDate":"2020-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/27/4b/nihms-1067860.PMC7051033.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"37699039","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Challenges In Conducting Research Studies In Arabic Countries","authors":"N. Sheblaq, Amal Al Najjar","doi":"10.2147/oajct.s215738","DOIUrl":"https://doi.org/10.2147/oajct.s215738","url":null,"abstract":"","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":" ","pages":""},"PeriodicalIF":1.2,"publicationDate":"2019-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/oajct.s215738","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47965001","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":"Verified Hepatorenal Syndrome Reversal As A Robust Multi-Component Primary End Point: The CONFIRM Study Trial Design","authors":"K. Jamil, S. Pappas, F. Wong, A. Sanyal","doi":"10.2147/oajct.s224974","DOIUrl":"https://doi.org/10.2147/oajct.s224974","url":null,"abstract":"Background: Hepatorenal syndrome type 1 (HRS-1) is an uncommon, rapidly progressing, potentially fatal renal failure if left untreated. Terlipressin is a vasopressin analog that is fi rst-line therapy in combination with albumin for treatment of HRS-1 in countries outside of North America. In two previous Phase III clinical trials, terlipressin showed favorable effects on improvement in renal function compared with placebo; however, neither study showed a signi fi cant between-group difference in the primary end point. CONFIRM (NCT02770716) is an ongoing clinical trial designed to address operational challenges observed in the previous studies by using a novel, more clinically relevant primary end point than the previous studies. Methods: This Phase III, randomized, double-blind, multicenter study is expected to enroll about 300 patients at approximately 70 sites in the US and Canada. Patients with cirrhosis and ascites demonstrating renal impairment via rapidly progressive worsening of serum creatinine (SCr) level ( ≥ 199 μ mol/L [ ≥ 2.25 mg/dL] with a trajectory of SCr doubling over 2 weeks) are randomized 2:1 to receive either terlipressin 1 mg every 6 hrs as an IV bolus injection or placebo. The design of the study is generally similar to previous terlipressin prospective studies in the setting of HRS-1. A key feature differentiating this study from the previous ones centers around a novel, multi-component ef fi cacy variable that extends beyond the traditional outcome of improvement in renal function to include durability of treatment-related effects on renal replacement therapy (RRT) requirements and survival. To meet criteria for the primary ef fi cacy end point in CONFIRM, patients must show not only HRS reversal con fi rmed by two SCr values ≤ 1.5 mg/dL, but also survive, without RRT, for at least 10 days after achieving it. Conclusion: Data from this pivotal study will demonstrate whether terlipressin treatment is effective as measured by a new, clinically meaningful, multi-component primary ef fi cacy end point.","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":" ","pages":""},"PeriodicalIF":1.2,"publicationDate":"2019-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/oajct.s224974","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45255010","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":"The European Medicines Agency Clinical Data Website Enables Insights Into Clinical Development Timelines And Strategy","authors":"S. Lehmann, R. Allard, Y. Boehler","doi":"10.2147/oajct.s205842","DOIUrl":"https://doi.org/10.2147/oajct.s205842","url":null,"abstract":"Sarah Lehmann 1,2 René Allard 2 Yvonne-Beatrice Boehler 1Faculty of Applied Natural Sciences, TH Koeln University of Applied Sciences, Leverkusen, Germany; 2Grünenthal Innovation, Drug Development, Data Sciences’ Grünenthal GmbH, Aachen, Germany Purpose: The clinical study report (CSR) documents of a full clinical development pathway (CDP) have been publicly available on the European Medicines Agency (EMA; Amsterdam, Netherlands) clinical data website (ECDW) since October 2016. Our analysis aimed to determine the extent to which the available clinical development program could be assessed. Methods: The documents available on the ECDW up to April 1, 2018 and the corresponding European Public Assessment Report (EPAR) were reviewed. Information extracted from the available CSRs focused on dates, phase of development, module leaf structure, and number of protocol amendments. Data analyses included generalized activity normalization time table (GANTT) charts and network analyses. Results: Of the 86 available CDPs, 55 were initial marketing authorizations covering a diverse range of clinical developments from generics to advanced therapy in the electronic common technical documents (eCTDs). Non-redacted dates were available in 444 CSRs from 15 CDPs to perform retrospective project clinical development management analyses. In these 15 marketing authorizations, the median timespan to submission was 9.3 years (range: 6.2–22.2). The timespan within these 15 clinical developments ranged from 5.9 to 21.4 years (median 8.3). The median time to first-subject-in in the first controlled clinical study pertinent to the claimed indication (CCSPCI) was 4.4 years (range: 0–12.1); the duration of the CCSPCI ranged from 2.4 to 16.9 years (median: 4.4; interquartile range: 4.2–7.0). Four CDPs had concurrent subject enrolment, while seven CDPs had seamless study designs. Subject participation ranged from 52% to 97% of a clinical development timeline. Conclusion: The publication of CSR documents by the EMA has enabled insights into timelines and project management aspects of the clinical development of medications.","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":" ","pages":""},"PeriodicalIF":1.2,"publicationDate":"2019-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/oajct.s205842","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"48443780","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":"An evaluation of informed consent comprehension by adult trial participants in South Africa at the time of providing consent for clinical trial participation and a review of the literature","authors":"L. Burgess, Berna Gerber, K. Coetzee, Marli Terblanche, Gareth Agar, T. Kotze","doi":"10.2147/OAJCT.S145068","DOIUrl":"https://doi.org/10.2147/OAJCT.S145068","url":null,"abstract":"Lesley Jean Burgess Berna Gerber Kathleen Coetzee Marli Terblanche Gareth Agar Theunis JvW Kotze 1TREAD Research CC, Cardiology Unit, Department of Medicine, Stellenbosch University and Tygerberg Hospital, Parow, South Africa; 2University of Liverpool/Laureate Online Education, Liverpool, UK; 3Division of SpeechLanguage and Hearing Therapy, Faculty of Medicine and Health Sciences, Stellenbosch University Stellenbosch, South Africa Introduction: The informed consent process is a fundamental part of clinical trials and is driven by both a legal and ethical agenda. The process may be seriously compromised if trial participants sign the informed consent document without fully understanding its contents. In developing countries such as South Africa, this concern is important due to the potential vulnerability of these patients and their risk for research exploitation. Aim: To evaluate the understanding of 11 important components and concepts related to clinical research by adult trial participants in a developing country at the time of providing consent for trial participation. Methods: 46 consecutive adult patients who qualified and consented to being enrolled in ongoing cardiovascular risk clinical trials at TREAD Research in the Western Cape, South Africa, were included in this study. After giving informed consent, participants were subjected to both a close-ended (self-report) and an open-ended method (descriptive narrative) to assess their understanding of various components and concepts related to clinical research pertaining to the initial informed consent document. The descriptive narrative was recorded and then later transcribed and assessed by two independent assessors. Results: There was a marked difference between the two methodologies used to assess patient comprehension of the various components. With the exception of concepts voluntariness and right to withdraw, trial participants’ understanding of the informed consent document was poor – especially with regard to the following concepts: randomization, risks, placebo and blinding. Higher levels of comprehension were obtained for the participant selfreports and lower levels for the narrative descriptions. Conclusion: The participant comprehension at this site was poor, and the process for taking informed consent subsequently needs to be modified so as to improve informed consent comprehension.","PeriodicalId":19500,"journal":{"name":"Open Access Journal of Clinical Trials","volume":"1 1","pages":""},"PeriodicalIF":1.2,"publicationDate":"2019-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.2147/OAJCT.S145068","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42604090","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}