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Journal of Surgical Oncology Jan 2022Surgeon-led clinical trials have defined the standard of care for locoregional pancreatic cancer to date. The infrastructure and collaborative nature of cooperative...
Surgeon-led clinical trials have defined the standard of care for locoregional pancreatic cancer to date. The infrastructure and collaborative nature of cooperative oncology groups offer many advantages, such as providing an ideal mechanism through which multidisciplinary pancreatic cancer trials are performed. As key members of the treatment team, surgeons bring experience and expertise to the design of surgical and multidisciplinary trials and are uniquely poised to be leaders of future pancreatic cancer trials.
Topics: Clinical Trials as Topic; Humans; Multicenter Studies as Topic; Neoplasm Staging; Pancreatic Neoplasms; Patient Care Team; Randomized Controlled Trials as Topic; Surgical Oncology
PubMed: 34586635
DOI: 10.1002/jso.26701 -
Oncology (Williston Park, N.Y.) Oct 2019The year 2019 has offered up many advances in immunotherapy. These advances shed light on such important aspects as biomarkers, better targeted therapies, chemotherapy...
The year 2019 has offered up many advances in immunotherapy. These advances shed light on such important aspects as biomarkers, better targeted therapies, chemotherapy outcomes, and resistance. Summarized herein are several high-profile conference presentations that highlight key advances in the field.
Topics: Antineoplastic Agents; Clinical Trials as Topic; Humans; Immunotherapy; Molecular Targeted Therapy; Neoplasms; Prognosis
PubMed: 31661150
DOI: No ID Found -
Urologic Oncology Mar 2021Clinical trials are pillars of modern clinical evidence generation. However, the clinical trial enterprise can be inefficient, and trials often fail before their planned...
OBJECTIVES
Clinical trials are pillars of modern clinical evidence generation. However, the clinical trial enterprise can be inefficient, and trials often fail before their planned endpoint is reached. We sought to estimate how often urologic oncology trials fail, why trials fail, and associations with trial failure.
METHODS
We queried phase 2/3 urologic clinical trial data from ClinicalTrials.gov registered between 2007 and 2019, with status marked as active, completed, or terminated. We extracted relevant trial data, including anticipated and actual accrual, from trial records and ClinicalTrials.gov archives. We manually coded reasons given in the "why stopped" free text field for trial failure into categories (poor accrual, interim results, toxicity/adverse events, study agent unavailable, canceled by the sponsor, inadequate budget, logistics, trial no longer needed, principal investigator left, no reason given, or other). We considered trials terminated for safety or efficacy to be completed trials. Trials marked as terminated for other reasons were considered failed trials. We then estimated the rate of trial failure using competing risks methods. Finally, we assessed associations with trial failure using a Cox proportional hazards model.
RESULTS
A total of 1,869 urologic oncology trials were included. Of these, 225 (12.0%) failed, and 51 (2.7%) were terminated for "good" reasons (e.g., toxicity, efficacy). Of the 225 failed trials, 122 (54%) failed due to poor accrual. Failed trials had a lower anticipated accrual than successfully completed trials (55 vs. 63 patients, P<0.001). A total of 6,832 patients were actually accrued to failed trials. The 10-year estimated risk of trial failure was 17% (95% CI 15%-22%). Single center trials, phase 3 trials, drug trials, and trials with exclusively USA sites were more likely to fail.
CONCLUSION
We estimate that 17%, or roughly 1 in 6, of urologic oncology trials fail, most frequently for poor accrual. Further investigations are needed into systemic, trial, and site-specific factors that may impact accrual and successful trial completion.
Topics: Clinical Trials as Topic; Early Termination of Clinical Trials; Humans; Urologic Neoplasms
PubMed: 33257221
DOI: 10.1016/j.urolonc.2020.10.070 -
Ending Gender Inequality in Cardiovascular Clinical Trial Leadership: JACC Review Topic of the Week.Journal of the American College of... Jun 2021Women are under-represented as leaders of cardiovascular randomized controlled trials, representing 1 in 10 lead authors of cardiovascular trials published in... (Review)
Review
Women are under-represented as leaders of cardiovascular randomized controlled trials, representing 1 in 10 lead authors of cardiovascular trials published in high-impact journals. Although the proportion of cardiovascular specialists who are women has increased in recent years, the proportion of cardiovascular clinical trialists who are women has not. This gap, underpinned by systemic sexism, has not been adequately addressed. The benefits of diverse randomized controlled trial leadership extend to patients and professionals. In this position statement, we present strategies adopted by some organizations to end gender inequality in research leadership. We offer an actionable roadmap for early-career researchers, scientists, academic institutions, professional societies, trial sponsors, and journals to follow, with the goal of harnessing the strength of women and under-represented groups as research leaders and facilitating a just culture in the cardiovascular clinical trial enterprise.
Topics: Cardiology; Clinical Trials as Topic; Female; Humans; Leadership; Male; Periodicals as Topic; Physicians, Women; Sexism; United States
PubMed: 34112322
DOI: 10.1016/j.jacc.2021.04.038 -
Fertility and Sterility Jan 2020Although chemotherapy is no complete substitute for psychotherapy in the treatment of impotence of the male patient, nevertheless, in cases where psychologic and/or...
Although chemotherapy is no complete substitute for psychotherapy in the treatment of impotence of the male patient, nevertheless, in cases where psychologic and/or somatic failure plays a role as etiologic agent then indeed chemotherapy is indicated. -T. Jakobovits.
Topics: Age of Onset; Clinical Trials as Topic; Health Knowledge, Attitudes, Practice; Humans; Hypogonadism; Male
PubMed: 32033739
DOI: 10.1016/j.fertnstert.2019.10.020 -
Epilepsy & Behavior : E&B Oct 2020On May 22-24, 2019, the 15th Antiepileptic Drug and Device (AEDD) Trials Conference was held, which focused on current issues related to AEDD development from... (Review)
Review
On May 22-24, 2019, the 15th Antiepileptic Drug and Device (AEDD) Trials Conference was held, which focused on current issues related to AEDD development from preclinical models to clinical prognostication. The conference featured regulatory agencies, academic laboratories, and healthcare companies involved in emerging epilepsy therapies and research. The program included discussions around funding and support for investigations in epilepsy and neurologic research, clinical trial design and integrated outcome measures for people with epilepsy, and drug development and upcoming disease-modifying therapies. Finally, the conference included updates from the preclinical, clinical, and device pipeline. Summaries of the talks are provided in this paper, with the various pipeline therapeutics in the listed tables to be outlined in a subsequent publication.
Topics: Animals; Anticonvulsants; Clinical Trials as Topic; Congresses as Topic; Device Approval; Drug Development; Epilepsy; Florida; Genetic Testing; Humans; Mass Screening; National Institute of Neurological Disorders and Stroke (U.S.); United States
PubMed: 32563052
DOI: 10.1016/j.yebeh.2020.107189 -
Annals of Allergy, Asthma & Immunology... Nov 2020Food allergy is a common condition that can have a significant impact on the quality of life of affected individuals and their caregivers. Recent years have witnessed an... (Review)
Review
OBJECTIVE
Food allergy is a common condition that can have a significant impact on the quality of life of affected individuals and their caregivers. Recent years have witnessed an increased effort to identify new treatments for food allergy. Here, we review the need to identify core outcomes for measurement in clinical trials of food allergy treatments.
DATA SOURCES
We reviewed the literature regarding core outcome set development, the important role that these play in prioritizing patient-relevant outcomes, and the potential for core outcomes to accelerate the path to product marketing by allowing prompt and reliable evidence synthesis after trial publication.
STUDY SELECTIONS
We reviewed recent clinical trials of food allergy treatments to understand which outcomes have previously been measured, and also reviewed available core outcome set initiatives for other allergic conditions to understand which other outcomes might be explored in future trials.
RESULTS
Clinical trials of food allergy treatments have largely focused on outcomes that are relevant to investigators and commercial investors, especially the threshold of reactivity and immunologic changes. Future trials should consider addressing patient-important outcomes and should report the experiences of both adult and child participants and their caregivers.
CONCLUSION
There is a pressing need for core outcome set development for food allergy treatment trials.
Topics: Clinical Trials as Topic; Desensitization, Immunologic; Food Hypersensitivity; Humans; Outcome Assessment, Health Care; Quality of Life; Treatment Outcome
PubMed: 32569834
DOI: 10.1016/j.anai.2020.06.023 -
Journal of Geriatric Oncology Jan 2023Conducting older adult-specific clinical trials can help overcome the lack of clinical evidence for older adults due to their underrepresentation in clinical trials.... (Review)
Review
INTRODUCTION
Conducting older adult-specific clinical trials can help overcome the lack of clinical evidence for older adults due to their underrepresentation in clinical trials. Understanding factors contributing to the successful completion of such trials can help trial sponsors and researchers prioritize studies and optimize study design. We aimed to develop a model that predicts trial failure among older adult-specific cancer clinical trials using trial-level factors.
MATERIALS AND METHODS
We identified phase 2-4 interventional cancer clinical trials that ended between 2008 and 2019 and had the minimum age limit of 60 years old or older using Aggregate Analysis of ClinicalTrials.gov data. We defined trial failure as closed early for reasons other than interim results or toxicity or completed with a sample of <85% of the targeted size. Candidate trial-level predictors were identified from a literature review. We evaluated eight types of machine learning algorithms to find the best model. Model fitting and testing were performed using 5-fold nested cross-validation. We evaluated the model performance using the area under receiver operating characteristic curve (AUROC).
RESULTS
Of 209 older adult-specific clinical trials, 87 were failed trials per the definition of trial failure. The model with the highest AUROC in the validation set was the least absolute shrinkage and selection operator (AUROC in the test set = 0.70; 95% confidence interval [CI]: 0.53, 0.86). Trial-level factors included in the best model were the study sponsor, the number of participating centers, the number of modalities, the level of restriction on performance score, study location, the number of arms, life expectancy restriction, and the number of target size. Among these factors, the number of centers (odds ratio [OR] = 0.83, 95% CI: 0.71, 0.94), study being in non-US only vs. US only (OR = 0.32, 95% CI: 0.12, 0.82), and life expectancy restriction (OR = 2.17, 95% CI: 1.04, 4.73) were significantly associated with the trial failure.
DISCUSSION
We identified trial-level factors predictive of trial failure among older adult-specific clinical trials and developed a prediction model that can help estimate the risk of failure before a study is conducted. The study findings could aid in the design and prioritization of future older adult-specific clinical trials.
Topics: Aged; Humans; Neoplasms; Clinical Trials as Topic; Treatment Outcome; Research Design
PubMed: 36437194
DOI: 10.1016/j.jgo.2022.11.003 -
Patient Education and Counseling Sep 2020Oncology clinical trials use a variety of clinical endpoints. Patients' understanding of the differences between clinical endpoints is important because misperceptions... (Review)
Review
OBJECTIVES
Oncology clinical trials use a variety of clinical endpoints. Patients' understanding of the differences between clinical endpoints is important because misperceptions of treatment efficacy may affect treatment decisions. The objective of this literature review is to find and synthesize available empirical publications assessing patients' understanding of common oncology clinical endpoints.
METHODS
We conducted a literature search of 5 databases and 3 conferences, limiting the search to articles and abstracts published in English through September 2018. We reviewed the titles and abstracts for inclusion, then reviewed full texts to determine if they reported empirical research studies focused on (1) clinical endpoints, (2) oncology, and (3) patient understanding. The original search identified 497publications, of which 13 met the inclusion criteria.
RESULTS
Available literature yields little information on this topic.The few publications that do exist suggest that healthcare professionals and cancer patients generally do not discuss clinical endpoint concepts and that patients can be confused about the purpose of a treatment based on misperceptions about endpoints.
CONCLUSIONS
Research is needed on how to discuss oncology clinical endpoints with patients.
PRACTICE IMPLICATIONS
Patient-friendly definitions of clinical endpoints may help healthcare providers communicate important information about treatments to patients.
Topics: Clinical Trials as Topic; Comprehension; Disease-Free Survival; Endpoint Determination; Humans; Medical Oncology; Neoplasms; Patient Education as Topic; Patients
PubMed: 32273145
DOI: 10.1016/j.pec.2020.03.018 -
Spinal Cord Sep 2019Traumatic spinal cord injury (SCI) leads to immediate neuronal and axonal damage at the focal injury site and triggers secondary pathologic series of events resulting... (Review)
Review
Traumatic spinal cord injury (SCI) leads to immediate neuronal and axonal damage at the focal injury site and triggers secondary pathologic series of events resulting in sensorimotor and autonomic dysfunction below the level of injury. Although there is no cure for SCI, neuroprotective and regenerative therapies show promising results at the preclinical stage. There is a pressing need to develop non-invasive outcome measures that can indicate whether a candidate therapeutic agent or a cocktail of therapeutic agents are positively altering the underlying disease processes. Recent conventional MRI studies have quantified spinal cord lesion characteristics and elucidated their relationship between severity of injury to clinical impairment and recovery. Next to the quantification of the primary cord damage, quantitative MRI measures of spinal cord (rostrocaudally to the lesion site) and brain integrity have demonstrated progressive and specific neurodegeneration of afferent and efferent neuronal pathways. MRI could therefore play a key role to ultimately uncover the relationship between clinical impairment/recovery and injury-induced neurodegenerative changes in the spinal cord and brain. Moreover, neuroimaging biomarkers hold promises to improve clinical trial design and efficiency through better patient stratification. The purpose of this narrative review is therefore to propose a guideline of clinically available MRI sequences and their derived neuroimaging biomarkers that have the potential to assess tissue damage at the macro- and microstructural level after SCI. In this piece, we make a recommendation for the use of key MRI sequences-both conventional and advanced-for clinical work-up and clinical trials.
Topics: Brain; Clinical Trials as Topic; Humans; Magnetic Resonance Imaging; Neuroimaging; Practice Guidelines as Topic; Spinal Cord Injuries
PubMed: 31267015
DOI: 10.1038/s41393-019-0309-x