The results of the DEEP-2 paediatric clinical trial are now available on ClinicalTrials.org as well as on the EU Clinical Trials Register. DEEP-2 is a multi-centre, randomised, open-label, non-inferiority active-controlled efficacy-safety trial involving paediatric patients from 1 month to less than 18 years affected by transfusion-dependent haemoglobinopathies (DEEP-2; EudraCT 2012–000353-31; NCT01825512) performed according to a Paediatric Investigation Plan (PIP) agreed by PDCO (P/0357/2016). The study was part of the EU funded project “DEferiprone Evaluation in Paediatrics” (DEEP, FP7 HEALTH-F4-2010-261483) coordinated by CVBF, also acting as a Sponsor of the study. The DEEP-2 Trial Leader, and CVBF CEO, Donato Bonifazi said that “The study showed that deferiprone was effective and safe in inducing control of iron overload during 12 months of treatment in paediatric patients with transfusion-dependent haemoglobinopathies. Considering the need for availability of more chelation treatments in paediatric populations, deferiprone offers a valuable treatment option for this age group.”
On 29 April 2021 the BMC Medical Ethics published the article "Ethical and procedural issues for applying researcher-driven multi-national paediatric clinical trials in and outside the European Union: the challenging experience of the DEEP project" written by the DEEP-2 Study trial leaders and Ethics board members. The article was aimed to evaluate the impact of the different local and national rules and procedures and of their complexity on the paediatric trial authorisation process in different EU and non-EU countries in terms of timelines for the final EC approval and CA authorisation from the preparation of submission package to the release of authorisation and approval. The main results shown that the authorisation process was completed in 7.7 to 53.8 months in European countries and in 17.1 to 27.1 months in non-European countries. The main factors influencing these timelines were the requests for changes/clarifications in European countries and the different national legislations in non-European countries. DEEP-2 is a multi-centre, randomised, open-label, non-inferiority active-controlled efficacy-safety trial involving paediatric patients from 1 month to less than 18 years affected by transfusion-dependent haemoglobinopathies (DEEP-2; EudraCT 2012–000353-31; NCT01825512) performed according to a Paediatric Investigation Plan (PIP) agreed by PDCO (P/0357/2016). The study is part of the EU funded project “DEferiprone Evaluation in Paediatrics” (DEEP, FP7 HEALTH-F4-2010-261483) coordinated by CVBF. The study was based on the setting up of a unique submission package to ECs and CAs based on GCP and other specific paediatric EU requirements, case by case adaptation of this package according to the national frameworks in force at the time of the submission in each involved country. Authors acknowledged the contribution given by TEDDY Network providing access to its regulatory database. Read the entire article here.
The European Joint Programme on Rare Diseases has launched its first academic education course “Diagnosing Rare Diseases: from the Clinic to Research and back” related to diagnosis in the rare disease’s context. The course will start on April 26th and will last 5 weeks with 3 hours weekly study. In 2019 the EJP RD has launched a survey, to which Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF) has participated, to examinate the needs, targets, and expectations around the development of an online academic training courses tackling rare diseases research. The development of the courses is coordinated by the Foundation for Rare Diseases, one of the EJP RD partners. Who is the course for? This course is designed for individuals with a keen interest in diagnostic research and rare diseases. While primarily designed for medical students and PhD/post-doc students in biomedical sciences, it will also be of interest to Patients Advocacy Organisations’ representatives, healthcare professionals or paramedics who want to further their knowledge of rare diseases diagnosis. What topics will you cover? The diagnostic process and the types of genetic tests available for rare diseases. The differences in rare genetic diseases patient pathways. Technological advances for diagnostic research. The role of collaborative studies and data sharing in rare diseases diagnosis. The impact of having or lacking a diagnosis on patients’ lives. The role and place of physiopathology approaches as well as social sciences research in the context of rare diseases diagnosis. Learners will gain insight into patients’ experiences, will discuss key issues relating to this topic and will also have the possibility to undertake their own internet-based investigations. To register for free and have more information on the course click here.
EMA has recently launched the CTIS training webpage for all user groups and organisations to facilitate their preparedness. This page contains information about the CTIS training programme and the online self-study material catalogue as foreseen by the CTIS training strategy. The material available will gradually increase as the catalogue is complete module by module in batches. The positive feedback received on the materials has been greatly facilitated by the engagement of experts, including sponsor experts on CTIS and training, in the material production through consultation on learning objectives and validation of materials. EMA’s training resources are tailored for clinical trial sponsors and staff of the European Union (EU) Member States, European Commission and other organisations who will use the system. On April 26th from 10.00 to 11.30 am EUCROF Events&Training working group, chaired by Donato Bonifazi, is organising the webinar on the EMA Clinical Trials Information System. In this webinar, Marieke Meulemans, CEO and Founder of GCP Central B.V., shows what training material has been created, where to find it and supports your preparation in the use of the CTIS with useful tips and considerations. Attending this webinar makes you aware of the steps to take in the next 9 months to get ready before the CTIS goes live in January 2022. More information is available here.
We are pleased to inform you that the article "Diagnosis and Treatment of Chronic Neuropathic and Mixed Pain in Children and Adolescents: Results of a Survey Study amongst Practitioners" has been published in Children open access journal. The article shows the results of a questionnaire administrated to investigate the international practice amongst practitioners for the diagnosis and treatment of chronic, neuropathic pain in children and adolescents. The questionnaire consisted of 33 questions divided in 3 sections. 117 returned questionnaires were analysed, of which 41 (35%) were fully completed and 76 (65%) were partially completed. Most respondents based the diagnosis of neuropathic pain on physical examination (68 (58.1%)), patient history (67 (57.3%)), and underlying disease (59 (50.4%)) combined. The main results showed that Gabapentin, amitriptyline, and pregabalin were the first-choice treatments for moderate neuropathic pain; Tramadol, ibuprofen, amitriptyline, and paracetamol were the first-choice treatments for moderate mixed pain; Consensus on the diagnostic process of neuropathic pain in children and adolescents is lacking. Drug treatment varies widely for moderate, severe neuropathic, and mixed pain. Hence, diagnostic tools and therapy need to be harmonized and validated for use in children. The GAPP project (GAbapentin in Paediatric Pain) was a FP7 funded project coordinated by Consorzio per Valutazioni Biologiche e Farmacologiche focused on the development of gabapentin for the treatment of chronic neuropathic and mixed pain in children, a condition where gabapentin, as demonstrated in adults, was expected to bring great benefit to children. The GAPP project has laid the groundwork for the clinical research in paediatric chronic pain. A large international scientific Consortium was set up, with experienced professionals in the field of pain that worked together with the purpose of aiding the design of two clinical [...]
On March 18th 2021, the European Medicine Agency (EMA) has published on its website the preliminary results on the evaluation of the AstraZeneca Covid-19 vaccine adverse events onset. EMA’s safety committee Pharmacovigilance Risk Assessment Committee (PRAC) has started an evaluation of cases of blood clots, some with unusual features such as low numbers of platelets, in recipients of COVID-19 Vaccine AstraZeneca. The evaluation was looking at the available data related to all thromboembolic events reported after vaccination. National agencies provided additional support to gather missing and incomplete information as quickly as possible, particularly where it relates to these unusual cases. The PRAC concluded its preliminary review and confirmed that: the benefits of the vaccine in combating the still widespread threat of COVID-19 (which itself results in clotting problems and may be fatal) continue to outweigh the risk of side effects; the vaccine is not associated with an increase in the overall risk of blood clots (thromboembolic events) in those who receive it; there is no evidence of a problem related to specific batches of the vaccine or to particular manufacturing sites; however, the vaccine may be associated with very rare cases of blood clots associated with thrombocytopenia, i.e. low levels of blood platelets (elements in the blood that help it to clot) with or without bleeding, including rare cases of clots in the vessels draining blood from the brain (CVST). Information for patients COVID-19 Vaccine AstraZeneca is not associated with an increased overall risk of blood clotting disorders. There have been very rare cases of unusual blood clots accompanied by low levels of blood platelets (components that help blood to clot) after vaccination. The reported cases were almost all in women under 55. Because COVID-19 [...]
The HMA-EMA Big Data Steering Group is pleased to announce the upcoming virtual workshop focused on “data characterization and discoverability” that will be held on Monday, 12 April 2021. Data characterization and discoverability are goals for the scientific community and for medicines regulation. The HMA-EMA joint Big Data Task Force recommended “to promote data discoverability through the identification of metadata” as part of its Recommendation III, which is reflected in the Big Data Steering Group work plan. The objective of this workshop is to review and gather stakeholders’ feedback on a: preliminary list of metadata required for characterising real-world data sources, and their definitions; proposed process to collect and maintain metadata from real-world data sources; proof-of-concept catalogue of data sources and metadata currently being developed. The workshop agenda is available here. More information on this project can be found on EU Big Data webpage. Photo by https://www.ema.europa.eu/
PedCRIN webinar series: An opportunity to discover our tools for the setup and management of paediatric and neonatal clinical trials
From April 7th to June 16th the PedCRIN project is launching a webinar series to share some of the most interesting outcomes of the project. The webinars are aimed to present the tools that have been developed to facilitate the execution of clinical trials for neonates, children and young people. On April 7th 2021, Viviana Giannuzzi from Gianni Benzi Foundation and Cristina Manfredi from CVBF, PedCRIN’s Partner, will explain the tools for the management of paediatric trials: handling biosamples and assessing causality of adverse events. You can register here. For more information visit the webinars dedicated webpage.
We are happy to announce you the cASPerCF has opened the first two sites participating in the Study and can now start the patient’s recruitment. The first Site Initiation Visit (SIV) was held on February 12th, 2021 at the Ospedale Pediatrico Bambino Gesù (OPBG) in Rome, followed by the second SIV held on March 9th, 2021 at the Azienda Socio Sanitaria Territoriale degli Spedali Civili in Brescia. cASPerCF study will test an antifungal drug called posaconazole in children and young people aged 8-17 years with Cystic Fibrosis and Aspergillus infection. cASPerCF is a non-industry proof of viability study performed under the c4c Project. The Sponsor of the Study is Ospedale Pediatrico Bambino Gesù (OPBG), Italy and the Coordinating Centre for the study is the Exeter Clinical Trials Unit located at the University of Exeter, UK. In this study CVBF is responsible for Sites Assessment, Data Management, Study Monitoring, Trial Master File preparation and maintenance.
Lo scorso 4 marzo si è tenuto il webinar di presentazione delle Factory dell’area Salute dell’uomo e dell’ambiente selezionate dall’intervento “Estrazione dei Talenti” per accompagnare i Team di aspiranti imprenditori innovatori nella valorizzazione dei loro progetti con percorsi personalizzati di accelerazione di impresa. A presentare la Factory EVOLUTIO START UP con un intervento è stato il Direttore dell’Istituto di Cristallografia – CNR, Dott. Michele Saviano che ha illustrato le peculiarità del partenariato coinvolto nonché i servizi offerti. EVOLUTIO START UP supporterà le idee innovative negli ambiti ricerca biomedicale e sviluppo farmaceutico, mediante percorsi di valorizzazione, azioni di trasferimento tecnologico e business development. Al termine della sessione mattutina, i rappresentanti di EVOLUTIO START UP, Sara Falvo (BIPCA – Bioindustry Park Silvano Fumero), Mariangela Lupo (CVBF – Consorzio per Valutazioni Biologiche e Farmacologiche) e Michele Saviano (CNR IC) hanno incontrato direttamente gli interessati durante i colloqui one-to-one. L’avviso Selezione Team, dell’intervento promosso da Regione Puglia e ARTI Estrazione dei Talenti, si rivolge ad aspiranti imprenditori innovativi, occupati o inoccupati – anche se beneficiari di strumenti di sostegno al reddito – che abbiano voglia di rendere concreto un progetto d’innovazione e ricevere i servizi di accompagnamento e tutorship di 300 ore forniti dalle Factory. Come candidarsi Possono candidarsi per accedere ai programmi proposti dalle Factory gruppi informali di aspiranti imprenditori e/o potenziali startupper (minimo tre persone) che condividono un’idea imprenditoriale ad alta intensità di conoscenza. L’avviso “Selezione Team” è attivo ed è organizzato a sportello: le candidature che pervengono sono valutate con cadenza bimestrale, fino all’esaurimento delle risorse disponibili.