Clinical Trials Update Research Articles

Clinical trials update 2015: Year in review.

This section of Headache annually reviews the status of recently completed and ongoing major clinical trials involving common headache disorders. The review will focus on multicenter trials of new therapies, as well as novel formulations of previously approved therapeutics. The Table summarizes the major therapeutic headache trials that were ongoing at the end of 2015, according to data obtained from both the "ClinicalTrials.Gov" website and from corporate press releases and presentations.

Recent Update of Clinical Drug Trials in Alzheimer’s Disease

Recent Update of Clinical Drug Trials in Alzheimer’s Disease

2015 Clinical trials update in sickle cell anemia.

Polymerization of HbS and cell sickling are the prime pathophysiological events in sickle cell disease (SCD). Over the last 30 years, a substantial understanding at the molecular level has been acquired on how a single amino acid change in the structure of the beta chain of hemoglobin leads to the explosive growth of the HbS polymer and the associated changes in red cell morphology. O2 tension and intracellular HbS concentration are the primary molecular drivers of this process, and are obvious targets for developing new therapies. However, polymerization and sickling are driving a complex network of associated cellular changes inside and outside of the erythrocyte, which become essential components of the inflammatory vasculopathy and result in a large range of potential acute and chronic organ damages. In these areas, a multitude of new targets for therapeutic developments have emerged, with several ongoing or planned new therapeutic interventions. This review outlines the key points of SCD pathophysiology as they relate to the development of new therapies, both at the pre-clinical and clinical levels.

September 2015 at a glance

Atrial and ventricular arrhythmias confer a substantial risk of mortality and morbidity in patients with heart failure (HF). The epidemiology, clinical impact and treatment of arrhythmias in HF are thoroughly described in a joint consensus document.1 Geographic differences in HF clinical trials are reviewed and discussed in two articles.2, 3 Another review regards the epidemiologic characteristics of HF in Asia,4 extending previous data from the same continent.5-8 Two articles concern acute HF. The first evaluates the prevalence of pulmonary edema in contemporary trials.9 The second regards the pathophysiology and prognostic impact of acute HF in a specific condition, the acute aortic syndrome.10 High heterogeneity exists in HF with preserved ejection fraction.11 A new classification is proposed based on the data from two major clinical trials and its potential to identify patients with a better response to treatment is shown.12 Two articles about cardiac resynchronization therapy (CRT) from MADIT-CRT regard its benefit in the patients without a history of advanced HF13 and its effects on arrhythmias.14 Other articles regard biomarkers, vasodilator treatment and a recent clinical trials update. Enjoy reading!

Clinical trials update from the European Society of Cardiology-Heart Failure meeting 2015: AUGMENT-HF, TITRATION, STOP-HF, HARMONIZE, LION HEART, MOOD-HF, and renin-angiotensin inhibitors in patients with heart and renal failure.

This article provides an overview on the key trials relevant to the pathophysiology, prevention, and treatment of heart failure (HF) presented at the Heart Failure Association (HFA) of the European Society of Cardiology (ESC) annual meeting held in Seville, Spain in May 2015. Trials reported include AUGMENT-AF (myocardial injections of calcium-alginate hydrogel), a propensity score-matched study of renin-angiotensin system antagonists in patients with HF and severe renal dysfunction, HARMONIZE (sodium zirconium cyclosilicate used to bind potassium), TITRATION, comparing two regimes for introducing LCZ696, STOP-HF, a trial of intramyocardial stromal cell-derived factor-1, MOOD-HF (escitalopram for patients with heart failure and depression), and LION HEART, a trial of intermittent levosimendan therapy. Unpublished reports should be considered as preliminary, since analyses may change in the final publication.

Clinical trial update: treatment of metastatic brain tumors using MRgFUS

Metastatic brain tumors are common lesions that are currently treated with multimodal therapy. Surgical resection, focused stereotactic radiosurgery, and whole brain radiation remain traditional therapies. It has been postulated that metastatic brain tumors could be treated effectively with MRgFUS using thermal ablation.

Systemic Therapy in Locally Advanced or Metastatic Adrenal Cancers: A Critical Appraisal and Clinical Trial Update

Mitotane and chemotherapy with etoposide, doxorubicin, and cisplatin plus mitotane (EDP-M) are the only therapies with demonstrated efficacy in advanced adrenocortical carcinoma. Prognostic and predictive factors are needed to identify patients who could obtain the best benefit from these treatments. Despite the strong rationale for their use, clinical trials on molecular targeted therapies have failed to demonstrate that these drugs are efficacious in the management of this extremely rare disease.

Update of Clinical Trials of Anti-PCSK9 Antibodies.

Hyperlipidemia is a predominant risk factor for cardiovascular disease (CVD). Statins have been successfully used to treat patients with dyslipidemia and decrease the events of CVD in addition to application of various other non-statin-lowering cholesterol agents, such as ezetimibe and niacin. However, there are still residual risks in patients with atherosclerotic CVD. Recently, proprotein convertase subtilisin/kexin type 9 (PCSK9), which was first identified in 2003, has been suggested to play an important role in the metabolism of low-density lipoprotein cholesterol (LDL-C). PCSK9 degrades the LDL-receptor, which may be pharmacologically targeted to improve the lipoprotein profile and future cardiovascular outcomes in patients with dyslipidemia. Several approaches to inhibiting PCSK9 activity have been theoretically proposed. Among them, monoclonal antibodies have been considered as the most promising strategy because of their large effect on lowering lipids as monotherapy and in combination with statins or ezetimibe. In this review, we mainly focus on the current status of monoclonal antibodies of PCSK9 and clinical trial results for an update on clinical application of monoclonal antibodies of PCSK9. The particular effects of monoclonal antibodies of PCSK9 on lipid profiles are also discussed.

Invited review: Stem cells and muscle diseases: advances in cell therapy strategies.

Despite considerable progress to increase our understanding of muscle genetics, pathophysiology, molecular and cellular partners involved in muscular dystrophies and muscle ageing, there is still a crucial need for effective treatments to counteract muscle degeneration and muscle wasting in such conditions. This review focuses on cell-based therapy for muscle diseases. We give an overview of the different parameters that have to be taken into account in such a therapeutic strategy, including the influence of muscle ageing, cell proliferation and migration capacities, as well as the translation of preclinical results in rodent into human clinical approaches. We describe recent advances in different types of human myogenic stem cells, with a particular emphasis on myoblasts but also on other candidate cells described so far [CD133+ cells, aldehyde dehydrogenase-positive cells (ALDH+), muscle-derived stem cells (MuStem), embryonic stem cells (ES) and induced pluripotent stem cells (iPS)]. Finally, we provide an update of ongoing clinical trials using cell therapy strategies.

Update of Therapeutic Clinical Trials for Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that is characterized by progressive death of motor neurons in the cortex, brainstem, and spinal cord. Until now, many treatment strategies have been tested in ALS, but so far only Riluzole has shown efficacy of slightly slowing disease progression. The pathophysiological mechanisms underlying ALS are multifactorial, with a complex interaction between genetic factors and molecular pathways. Other motor neuron disease such as spinal muscular atrophy (SMA) and spinobulbar muscular atrophy (SBMA) are also progressive neurodegenerative disease with loss of motor neuron as ALS. This common thread of motor neuron loss has provided a target for the development of therapies for these motor neuron diseases. A better understanding of these pathogenic mechanisms and the potential pathological relationship between the various cellular processes have suggested novel therapeutic approaches, including stem cell and genetics-based strategies, providing hope for feasible treatment of ALS. (Korean J Clin Neurophysiol 2015;17:1-16)

European Society of Cardiology (ESC) Congress Report from London 2015.

The Annual Congress of the European Society of Cardiology (ESC) was held in London from 29 August to 2 September 2015. It is the leading conference in cardiology in the world, with presentations on the latest scientific discoveries, innovations, technology, education, and clinical practices. More than 32,000 delegates and 5,000 exhibitors from 140 countries participated, sharing a number of scientific presentations, including 28 clinical hot lines, 18 clinical trial updates, 20 registry studies, 12 basic and translational science hot line studies, and 4,533 abstract studies. Japan had the highest number of accepted abstracts at the Congress, indicating the great contribution of Japanese scientists and the Japanese Circulation Society.

A Selection of Hot Topics in Ophthalmology

This article discusses current developments and trends in a variety of areas of ophthalmology. Specific topics include King-Devick test to identify concussion in athletes, immunoglobulin G4—related disease of the orbit, SMILE laser surgery, small-aperture implants, screening for retinopathy of prematurity, diabetic macular edema, as well as clinical trial updates.

Clinical trials update from the European Society of Cardiology meeting 2014: PARADIGM-HF, CONFIRM-HF, SIGNIFY, atrial fibrillation, beta-blockers and heart failure, and vagal stimulation in heart failure.

This article provides an overview of trials relevant to the pathophysiology, prevention, and treatment of heart failure, presented at the European Society of Cardiology meeting held in Barcelona in autumn 2014. Trials reported here include PARADIGM-HF (LCZ696 versus enalapril in heart failure), CONFIRM-HF (treatment of iron deficiency in heart failure), and SIGNIFY (ivabradine in patients with stable coronary artery disease). In addition, we discuss recent developments in the treatment of atrial fibrillation and the lack of benefit with the use of beta-blockers in these patients. Finally, the article describes recent advances in the use of vagal stimulation in patients with heart failure. © 2014 The Authors. ESC Heart Failure published by John Wiley & Sons Ltd on behalf of the European Society of Cardiology.

Red Face and its Relationship to Rosacea

1. Maria Vinall 1. Frank C. Powell, MD 1. University College Dublin, Dublin, Ireland Rosacea, a chronic inflammatory skin disease of unknown etiology, occurs mainly in red-haired, blue-eyed, fair-skinned individuals of Northern European and Celtic origin. This article discusses the association of rosacea with the Irish population, the clinical symptoms of rosacea, the management of red face symptoms, the diagnosis and management of red acne, as well as related clinical trial updates.

Hotline update of clinical trials and registries presented at the American College of Cardiology Congress 2014

This article provides information and commentaries on trials which were presented at the Hotline and Clinical Trial Update Sessions during the Late Breaking Clinical Trial Sessions at the 63rd annual meeting of the American College of Cardiology in Washington, USA, from 29th to 31st March 2014. This article gives an overview on a number of novel clinical trials in the field of cardiovascular medicine, which were presented. Comprehensive summaries have been generated from the oral presentation and the webcasts of the American College of Cardiology, similar to as previously reported and should provide the readers with the most comprehensive information of relevant publications. The discussed studies are US CoreValve, Choice, Symplcity-HTN-3, GRS, ZEUS, GIPS-III, HEAT-PPCI, COPR-2, MSC-HF, POISE-2, SIRS. The data were presented by leading experts in the field.

Congress of European society of cardiology (amsterdam, 2013): new recommendations and most important clinical studies

In this review we present short report on European Congress of Cardiology which took place in Amsterdam from August 3 to September 4, 2013. Four new European recommendations on the treatment of cardiovascular diseases are briefly characterized and reports of Hot Line and Clinical Trial Update scientific sessions are presented.

Clinical Trials Update - 2013: Year in Review

Letter to the Editor Clinical Trials Update – 2013: Year in Review Marcelo E. Bigal MD, PhD, Marcelo E. Bigal MD, PhD Chief Medical Officer Labrys Biologics, Inc., Doylestown, PA, USASearch for more papers by this author Marcelo E. Bigal MD, PhD, Marcelo E. Bigal MD, PhD Chief Medical Officer Labrys Biologics, Inc., Doylestown, PA, USASearch for more papers by this author First published: 02 April 2014 https://doi.org/10.1111/head.12313 Conflict of Interest: Dr. Bigal is a full-time employee of Labrys Biologics. Read the full textAboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onFacebookTwitterLinkedInRedditWechat No abstract is available for this article. Volume54, Issue4April 2014Pages 756-756 RelatedInformation

A report from the European Society of Cardiology Congress 2014 (August 30-September 3 - Barcelona, Spain).

The European Society of Cardiology (ESC) Annual Congress is the largest cardiology conference in the world and this year ran in Barcelona from August 30 to September 3. During the meeting, more than 30,000 cardiologists from over 100 countries met to share their knowledge in all cardiovascular fields, from basic science to management and prevention of cardiovascular diseases. Apart from more than 4,500 interesting abstracts presented in posters and oral sessions, five new ESC Clinical Practice Guidelines were presented among the latest clinical trial results, updates and registries.

European Society of Cardiology (ESC) congress report from Barcelona 2014.

The Annual Congress of the European Society of Cardiology (ESC) was held in Barcelona from 30th August to 3rd September 2014. More than 30,300 attendees from around the world shared the latest original research, including 27 clinical Hot Line studies, 12 basic science Hot Lines, 15 clinical trial updates, 19 registry studies, and 4,597 abstracts. Many important issues were presented, including novel treatment strategies for heart failure, acute coronary syndrome, interventional treatment for structural heart disease, renal denervation, novel anticoagulant therapies, atrial fibrillation and so on. In addition, 5 new ESC clinical practice guidelines (ie, myocardial revascularization, non-cardiac surgery, acute pulmonary embolism, hypertrophic cardiomyopathy, and aortic disease) were launched. It should be noted that Japan has recently been ranked in the top position in terms of the number of abstract submissions. Based on these activities, the ESC Congress has been recognized as the dominant scientific and educational forum for healthcare professionals in cardiology. We report the highlights and several key presentations of the ESC Congress 2014. The scientific activities and growing contributions of Japanese cardiologists or cardiovascular surgeons enhance the favorable relationship between the ESC and the Japanese Circulation Society.

Clinical Trials Update. 2013: Year in Review

This section of Headache annually reviews the status of recently completed and ongoing major clinical trials involving common headache disorders. The review will focus on multicenter trials of new therapies, as well as novel formulations of previously approved therapeutics. Table 1 summarizes the major therapeutic headache trials that are ongoing at the present time, according to data obtained from both the "ClinicalTrials.Gov" website and from corporate press releases and presentations.