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Agency: Cordis | Branch: H2020 | Program: RIA | Phase: PHC-13-2014 | Award Amount: 21.24M | Year: 2015

Cystic fibrosis (CF) is a progressive life-shortening disease caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene leading to a dysfunctional CFTR protein. The disease affects over 70,000 patients worldwide and while many mutations are known, the F508del mutation affects 90% of all patients. The absence of CFTR in the plasma membrane leads to a dramatic decrease in chloride efflux, resulting in viscous mucus that causes severe symptoms in vital organs like the lungs and intestines. For CF patients that suffer from the life threatening F508del mutation only palliative treatment exist. PRO-CF-MED addresses the specific challenge of this call by introducing the first disease modifying medication for the treatment of the CF patients with F508del mutation. The PRO-CF-MED project has been designed to assess the potential clinical efficacy of QR-010, an innovative disease modifying oligonucleotide-based treatment for F508del patients. Partners within PRO-CF-MED have generated very promising preclinical evidence for QR-010 which allows for further clinical assessment of QR-010 in clinical trials. PRO-CF-MED will enable the fast translation of QR-010 towards clinical practice and market authorisation. PRO-CF-MED has the potential to transform this life-threatening condition into a manageable one.

Agency: Cordis | Branch: H2020 | Program: RIA | Phase: PHC-16-2015 | Award Amount: 5.97M | Year: 2016

Cardiovascular diseases including myocardial infarction (MI), which entails the irreversible loss of heart muscle tissue, constitute a major socio-economic burden in global healthcare. With whole organ transplantation as the only treatment option for end-stage heart failure, MI patients could particularly benefit from advanced cell therapies aimed at the functional reconstitution of damaged hearts. Human induced pluripotent stem cells (hiPSCs) can be derived by reprogramming patients somatic cells. In contrast to adult (stem) cells e.g. from blood, bone marrow or the heart, hiPSCs have unlimited expandability and differentiation potential into all relevant cell types including cardiomyocytes, endothelial cells, pericytes and connective tissue-forming cells, making them highly attractive as a universal cell source for organ repair. However, technologies for the robust therapeutic scale production of hiPSC-derived progenies in line with GMP standards and at reasonable cost are currently lacking. TECHNOBEATs ultimate objectives are 1) to advance therapeutic scale cell production through innovative bioreactor technologies and novel cell monitoring tools, and 2) to develop regulatory compliant bioprocessing of innovative iPSC-based cardiac -tissue. The clinical translation of cardiac -tissue will require 3) the development and application of tools for improved cell delivery and longitudinal in vivo monitoring of cell grafts, and 4) proof-of-concept for safety and functional integration in physiologically relevant preclinical models of cellular heart repair. Through its interdisciplinary excellence, TECHNOBEATs consortium of leading European stem cell researchers, clinicians, tissue-, bioprocess-, and technical- engineers in industry and academia is ideally positioned to address these ambitious objectives. It will provide new treatment options for suffering patients and increase Europes attractiveness as a hub for innovative medical technologies.

Agency: Cordis | Branch: H2020 | Program: RIA | Phase: PHC-17-2014 | Award Amount: 6.65M | Year: 2015

Most older adults have multiple chronic diseases (multimorbidity) and multiple medications (polypharmacy). However, multimorbid patients are often excluded from clinical trials and most guidelines address diseases in isolation. Inappropriate drug prescription and poor drug compliance are common and contribute to up to 30% of hospital admissions. OPERAM investigators developed STOPP/START criteria to detect inappropriate drug use, both over- and underuse. Applying these criteria limits unnecessary polypharmacy and reduces underuse of indicated medications, but it remains uncertain whether systematic pharmacotherapy optimisation can improve clinical outcomes and reduce costs.We propose a multicentre randomised controlled trial to assess the impact of a userfriendly software-assisted intervention to optimise pharmacotherapy and to enhance compliance in 1900 multimorbid patients aged 75 years. Outcomes will include drug-related hospital admissions, health care utilisation, quality of life, patient preferences and cost-effectiveness. We will also perform several network meta-analyses (NMA) to provide new comparative evidence on the most effective and safest pharmacological and non-pharmacological interventions to reduce common causes of preventable hospital admissions (e.g. falls, fractures, bleeding). Therapy optimisation in the multimorbid elderly, enhanced compliance and discontinuation of less effective interventions have the potential to improve clinical, quality of life and safety outcomes, while reducing costs. We will provide a structured method with practical software solutions for optimal prescribing and new comparative evidence from NMAs for addressing multimorbidity and polypharmacy by means of customised, patient-centred guidelines. OPERAM ultimately aims at better healthcare delivery in primary and hospital care, based on effective, safe, personalised and cost-effective interventions that can be applied to the rapidly growing older population in Europe.

Agency: Cordis | Branch: H2020 | Program: RIA | Phase: PHC-17-2014 | Award Amount: 5.99M | Year: 2015

Every year, 1.3 million Europeans have a stroke and one million ultimately die of stroke. One third of stroke patients remain dependent on the help of others. The annual costs for stroke care in Europe are estimated at 64.1 billion. Stroke incidence increases almost exponentially with age, and the personal, societal, and economic burden of stroke is therefore largely driven by its frequent occurrence in the elderly. The elderly have been strongly underrepresented in previous stroke trials and treatment guidelines have no recommendations specific to this important group. Elderly patients are at the highest risk of complications after stroke, such as infections, fever, and dysphagia. These complications are strongly and independently associated with a higher risk of death or dependency. We will perform a pragmatic, randomised, open clinical trial with blinded outcome assessment in 3800 patients with acute stroke aged 66 years or older, to assess whether pharmacological prevention of infections and fever, and early management of dysphagia, will reduce the risk of death, poor functional outcome, and poor quality of life, and lead to reductions in the costs of stroke care throughout Europe. Patients will be randomised using a factorial design to preventive treatment for 4 days with ceftriaxone, paracetamol, and/or metoclopramide, or to standard care alone. The primary outcome is functional outcome at 3 months, assessed with the modified Rankin Scale (mRS), and analysed with ordinal logistic regression. The study will have 90% power to detect a statistically significant shift towards a favourable outcome, assuming a 5% absolute increase in the proportion of patients with a good outcome (mRS 0 to 2) in the intervention group, compared with controls. This simple, safe, and generally available treatment strategy has the potential to lead to an annual reduction of over 25 000 elderly Europeans being dead or dependent as a result of stroke, at very low costs.

Agency: Cordis | Branch: H2020 | Program: RIA | Phase: PHC-18-2015 | Award Amount: 6.31M | Year: 2016

Neonatal hypoxic-ischemic encephalopathy (HIE) is a major cause of death or long-term disability in infants born at term in the western world, affecting about 1-4 per 1.000 life births and consequently about 5-20.000 infants per year in Europe. Hypothermic treatment became the only established therapy to improve outcome after perinatal hypoxic-ischemic insults. Despite hypothermia and neonatal intensive care, 45-50% of affected children die or suffer from long-term neurodevelopmental impairment. Additional neuroprotective interventions, beside hypothermia, are warranted to further improve their outcome. Allopurinol is a xanthine oxidase inhibitor and reduces the production of oxygen radicals and brain damage in experimental, animal, and early human studies of ischemia and reperfusion. This project aims to evaluate the efficacy and safety of allopurinol administered immediately after birth to near-term infants with HIE in addition to hypothermic treatment. Beyond this primary objective, the project will provide information on the effect of hypothermia on pharmacokinetics of drugs with a similar metabolism as allopurinol in neonates. Furthermore it will give the opportunity to further develop and validate biomarkers for neonatal brain injury using advanced magnetic resonance imaging, biochemistry, and electroencephalogramms, which will then be available for future studies testing neuroprotective interventions. Finally, this trial will extend our knowledge about incidence of and risk factors for perinatal asphyxia and HIE possibly enabling generation of more preventive strategies for the future.

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