VISION-DMD (Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy, DMD) is a Phase 2a and 2b randomised, double-blind, parallel placebo- and active-controlled study. It will assess the safety and toxicity (phase 2a) and safety and efficacy (phase 2b) of the orphan drug VBP15 in ambulatory boys with Duchenne muscular dystrophy (DMD), a rare recessive X-linked form of muscular dystrophy. Funded by Horizon 2020 and the sponsor, Reveragen Biopharma Limited, the project aims to advance clinical development of VBP15 as a new therapy to revolutionise care for all patients with DMD by 2020. The study will be carried out over 18 to 24 months in approximately 15 countries and 30 international study sites. The phase 2a study will be conducted at approximately 10 U.S. study sites. The phase 2b study, supported by ECRIN, will be conducted at approximately 30 international study sites in 14 different countries (UK, France, Italy, Spain, Belgium, Netherlands, Denmark, Sweden, Switzerland, Poland, Czech Republic, Turkey, Israel, and Australia).

The trial is ongoing but ECRIN's involvement ended in 2019. 

Population: paediatric

Therapeutic hypothermia is recommended for unconscious survivors of out-of-hospital cardiac arrest. Are there differences in mortality, neurological function and adverse events between a target temperature management at 33°C and 36°C for 24 hours after cardiac arrest?

Population: adult | Intervention type: therapeutic medicinal product

Patients with blood poisoning - sepsis - often receive blood transfusions in the intensive care unit. The evidence that blood transfusion leads to improved outcome is limited and the blood may be harmful to some of these patients. To bridge the gap between clinical practice and evidence, a large randomised clinical trial is needed to document the efficacy and safety of RBC transfusion in these very sick patients.

Population: adult | Intervention type: therapeutic medicinal product

Huntington's disease is an autosomal dominant disease characterised by movement disorders and cognitive and neuropsychiatric disturbances. The clinical features usually emerge between 30 and 50 years of age and there is currently no curative treatment. Brain energy deficiency plays an important role in the pathophysiology of the disease. This study asks whether the administration of triheptanoin can effectively improve Huntington's disease as assessed by MRI, in vivo spectroscopy and clinical evaluation.

Population: adult | Intervention type: therapeutic medicinal product

There are currently no cures for Parkinson's disease (PD) but one of the most effective reparative therapies in patients to date has been with allotransplants of dopamine (DA) neuroblasts obtained from fetal ventral mesencephalic (VM) tissue. However, this cell transplantation approach has given inconsistent results, with some patients doing extremely well and coming off anti-PD medication for years, whilst others have shown no or only modest clinical improvements, and in some cases also developed severe, off-state graft-induced dyskinesias (GIDs). The reasons behind this heterogeneity of outcomes, and the emergence of GIDs in particular, need to be better understood, not least in the perspective of the rapid advances that are now being made in the development of stem-cell based therapies. There is therefore an urgent need to revisit the trials that have already been done with fetal VM tissue in PD patients, with the expectation that a critical reassessment can form the basis for an optimised and more standardised procedure that will translate into more consistently efficacious transplants with minimal side-effects. Over the last two years a group of international experts, including the key investigators of the previous European and North American trials, has been re-examining the outcome of these trials as well as reviewing the results obtained from recent and ongoing animal experimental studies, and identified a number of weaknesses that may explain the inconsistent outcome in previous trials. As a result of these discussions, the group has agreed to join forces in a new round of experimental work and cell therapy trials in PD, based on a new jointly developed protocol where all these factors are taken into account. In the first instance fetal VM tissue containing mesencephalic DA neuroblasts will be used, with the expectation that this will pave the way for bigger trials using dopaminergic neurons derived from stem cells.

Population: adult | Intervention type: therapeutic medicinal product

The aim of the TINN2 study is to evaluate the efficacy of azithromycin in prevention of bronchopulmonary dysplasia in preterm neonates.

The aim of TINN2 is to evaluate azithromycin, included in the EMEA priority list of the therapeutic areas that need specific drug evaluation in preterm and term neonates. Azithromycin is a macrolid antibiotic with anti-inflammatory properties active against Ureaplasma. It might be effective in reducing the severity of bronhopulmonary disease in which Ureaplasma infection and inflammation play a role. TINN2 involves European leaders in neonatology, paediatric pharmacology, methodology and SMEs that will establish links with ethical bodies and regulatory authorities. The programme will perform in silico experiments and evaluate formulations for neonates.

The randomized placebo-controlled trial will be optimized using age-appropriate state-of-the-art methods adapted to neonates (including in silico experiments, pharmacokinetics and pharmacogenetics) to validate the components of a Paediatric Investigation Plan. It will be performed by neonatologists trained in paediatric pharmacology and clinical research in line with guidelines on Good Clinical Practice. All the ethical issues will be considered, including pain and distress, blood sampling (number and volume) and informed consent.

Parent information sheets and consent form will be submitted to parents’ associations for approval. TINN2 will include short term safety and potential for long term adverse reactions. Results will be reported in order to allow a PUMA application and to improve neonatal care. Therefore, TINN2 will validate the appropriate use of azithromycin in neonates which will be of direct benefit to children, their families and health professionals. TINN2 will strengthen paediatric drug evaluation across Europe, support recent initiatives from the European pharmaceutical industry and build up a network of units with experience in clinical research that will be used for additional drug evaluation in neonates.

Intervention type: therapeutic medicinal product

This study is designed to determine whether micafungin is as efficacious as the current standard of fluconazole, to compare the safety of the two drugs in the treatment of proven neonatal candidiasis.

It is also designed to further elucidate the pharmacokinetics of the two products in the growing and developing neonate and premature infant.

Intervention type: therapeutic medicinal product

Ivermectin is currently the best drug to cure strongyloidiasis, but the standard single dose of 200 mcg/kg is probably not enough to guarantee a cure. As strongyloidiasis can be fatal in immunosuppressed patients, it is mandatory to define the optimal dosage to eradicate the parasite. The aim of this study is to define the most effective dose schedule of ivermectin to cure strongyloidiasis.

Population: adult | Intervention type: therapeutic medicinal product

The purpose of this study is to evaluate the efficacy of a polypill strategy containing aspirin (100 mg), ramipril (2.5, 5 or 10 mgs), and atorvastatin (40 mgs) compared with the standard of care (usual care according to the local clinical practices at each participating country) in secondary prevention of major cardiovascular events (cardiovascular death, nonfatal myocardial infarction, nonfatal ischemic stroke, and urgent revascularization) in elderly patients with a recent myocardial infarction.

Population: adult | Intervention type: therapeutic medicinal product

As the number of older people in Europe grows, increasing healthy life years is a priority. Cognitive decline, dementia (e.g. Alzheimer’s disease, AD), sleep disturbances and depression, all related to psychological distress and anxiety, are significant drivers of reduced quality of life in older adults. This study is part of a project that builds on evidence that lifestyle factors and meditation practice have the potential to downregulate these adverse factors and positively impact mental and neurological conditions including AD. In particular, the study aims to assess the short-term effects of an eight-week meditation intervention (versus cognitive training) in patients with subjective cognitive decline at risk for AD on behavioural measures including anxiety and wellbeing.

Population: adult | Intervention type: therapeutic medicinal product