European citizens affected by COVID-19 have been well-served by landmark clinical trials in hospitals that have found treatments that save many lives. However, there are fewer opportunities for people in the community to contribute to the urgent mission of finding treatments that speed recovery, and reduce the need for hospital admission in the first place. Desperately needed, evidence-based therapeutics for use in primary care have the potential for considerable reach and impact on individual suffering and functioning, as well as on the sustainability of health services. ECRAID-PRIME, a European Adaptive Platform Trial (APT) of COVID-19 therapeutics in primary care will build on many years of EU investment in infrastructure for primary care trials and a mature primary care research network that has pioneered novel, efficient, platform clinical trial designs. ECRAID-PRIME, with a focus on early phase studies of safety and efficacy of exciting candidate treatments for COVID-19 will be rapidly set up. Its goal is to test at least four strong candidate treatments, identifying suitability for inclusion in the next phases of research, and so leading to critical additions to the primary care therapeutic armamentarium against COVID-19. ECRAID-PRIME’s primary care focus will help complete the vison for a lasting, integrated, comprehensive and sustainable European clinical research preparedness and response capacity, ECRAID, that will provide a full, integrated suite of international priority clinical trials in intensive care units, hospital wards, and now in primary care.
Partners: University Medical Center Freiburg, MEDACTA INTERNATIONAL SA, Galeazzi orthopedic institute, University of Basel, ECRIN, University of Würzburg, MEFZG, FHG
Spontaneous healing of articular cartilage injuries is poor and untreated defects predispose to osteoarthritis. Current therapies, including innovative autologous cell-based treatments, cannot predictably and reproducibly restore cartilage structure and function. BIOCHIP will carry out a multicenter, prospective phase II clinical trial to treat knee cartilage injuries using engineered grafts based on autologous nasal chondrocytes (NC). As compared to typically employed articular chondrocytes, NC have a higher and more reproducible capacity to generate mature cartilage tissues. Importantly, molecular/mechanical characterization, large size animal studies and a phase I trial carried out by BIOCHIP partners have already shown the compatibility of NC upon implantation in a joint, with promising preliminary clinical results. BIO-CHIP’s specific objectives are: (1) To test the hypothesis that the maturation of NC-based cartilage grafts improves the clinical efficacy in the treatment of cartilage lesions (108 patients will be recruited to reach statistical significance) (2) To extend the range of clinical indications of NC-based grafts to so far untreatable pre-osteoarthritic lesions (‘kissing’ cartilage lesions in a sheep model) BIO-CHIP capitalizes on clinical experience of 4 reference centers for cartilage surgery, on established GMP manufacturing capacity and on preparation for commercial exploitation by a strong orthopedic device company. Demonstration of therapeutic efficacy of the new treatment will address a large clinical need (over 2 million cartilage defects/year worldwide), improve quality of life (reduce pain & disability in the young, delay prosthetic implants in the elderly), exploit a commercial opportunity (prospected revenues of up to 130 million €/year) and reduce healthcare costs (estimated 12,000€ healthcare savings/procedure). BIO-CHIP will consolidate the currently leading role of Europe in the development of cell-based cartilage regeneration strate
VISION-DMD aims to advance clinical development of the orphan drug VBP15 as a new therapy to revolutionise care for all patients with Duchenne muscular dystrophy (DMD) by 2020, in line with IRDiRC goals. DMD is an incurable, rare muscle wasting disease; boys progressively weaken, lose ambulation and death occurs by early adulthood. Corticosteroids (CS) are widely recognised to increase muscle strength and delay disease progression but global acceptance as standard of care is very variable due to severe side effects. VBP15 is an innovative steroid-like drug designed to retain or better CS efficacy and improve membrane stabilization with reduced or no side effects. VBP15 will increase the therapeutic window to slow disease progression and improve quality of life and lifespan for all DMD patients. Building on positive preclinical and Phase 1 results funded by government grants and international patient groups and based on FDA and EMA advice, VISION-DMD proposes a Phase 2 registration directed clinical programme aimed at an affordable therapy: Phase 2a will study the safety and tolerability of ascending doses of VBP15 in ambulant DMD boys; Phase 2b will demonstrate the efficacy and safety of two doses of VBP15 in young ambulant DMD boys. Both studies will be followed by extension studies for long term safety and efficacy data collection leading to cumulative exposure of up to 2100 drug months. The project proposes the Time to Stand Test as a highly relevant and reliable primary endpoint. Innovative exploratory serum biomarkers and novel wide scale MRI techniques will be used to investigate the VBP15 pharmacodynamics and the effect on muscle cellular pathology. VBP15 will meet the unmet need for better treatment for DMD with widespread acceptance and potentially be used in combination with stratified therapies as they are developed. The Consortium links the leading networks TREAT-NMD and CINRG with ECRIN-ERIC, for trial delivery and regulatory undertakings in Europe/US
The eXtreme DataCloud (XDC) project will develop scalable technologies for federating storage resources and managing data in highly distributed computing environments. The services provided will be capable of operating at the unprecedented scale required by the most demanding, data intensive, research experiments in Europe and Worldwide. XDC will be based on existing tools (TRL8+) that the project will enrich with new functionalities and plugins already available as prototypes (TRL6). The targeted platforms are the current and next generation e-Infrastructures deployed in Europe, such as the European Open Science Cloud (EOSC), the European Grid Infrastructure (EGI), the Worldwide LHC Computing Grid (WLCG) and the computing infrastructures that will be funded by the upcoming H2020 EINFRA-12 call. The main high-level topics addressed by the project include: federation of storage resources with standard protocols, smart caching solutions, policy driven data management based on Quality of Service, data lifecycle management, metadata handling and manipulation, data preprocessing and encryption during ingestion, optimized data management based on access patterns. All the developments will be driven and tested against real life use cases provided by the consortium partners representing research communities belonging to a variety of scientific domains: Life Science, Astrophysics, High Energy Physics, Photon Science and Clinical Research. The XDC software will be released as Open Source platforms available for general exploitation.
Partners: Jagiellonian University, ASTIR, IRFMN, UPJS, SB MARIBOR, OROBIX LIFE, ECRIN, FBK, ADMINISTRATION OF HEALTH REGION A.H.RE 7TH HEALTH REGION CRETE
To date, the possibility of doing research on quality-of-care assessment in emergency medicine has clashed against sustainability problems. The vast number of patients visiting an ED and the staff shortage that often afflicts these departments make ad hoc data collections unattainable. The only way to fill the gap between the need for clinical research and the availability of robust data is to directly extract such data from the EDs electronic health records (EHRs), avoiding dedicated data collection. Achieving this goal would enable distributed clinical research, which is now too much restricted to academic centres, and allow to leverage of clinical information to address a multitude of research questions. Nonetheless, obtaining consistent data from EHRs is a complex task. While a small part of the data registered in EHRs is structured (such as lab test results and vital parameters), most of the useful information on patients' conditions is variably contained in free text (e.g. presence of signs and symptoms, suspected and confirmed diagnosis, anamnesis, etc.). Moreover, as a proactive follow-up of ED patients is unfeasible, relying on the existing data sources is also necessary to measure the outcome of the patients at the most appropriate time interval for the research question of interest. This proposal has three main aims: 1) to develop new technical solutions to extract reliable clinical information from structured and unstructured data contained in different electronic patient files; 2) to FAIRify (i.e. making data Findable, Accessible, Interoperable, and Re-usable) the established databases for clinicians, researchers, health policymakers and citizens while respecting the European and national legislations; 3) to pilot the exploitation of the established databases in two relevant use cases: i) assessment of ED propensity to hospitalise a patient, and ii) development of a dashboard to be used by citizens and policymakers to improve the quality of care in ED.