The surfactant dysfunction disorders are an important group of molecularly and histologically defined entities, belonging to the interstitial lung diseases in children (chILD). Mutations in genes encoding for surfactant protein C, surfactant protein B, the lipid transporter ABCA3, thyroid transcription factor 1 and others, cause a significant group of phenotypically similar chronic interstitial lung diseases with high morbidity and early mortality.

Unfortunately no successfully proven treatments are available. Beyond oxygen supplementation, nutritional support and immunizations, the small molecule hydroxychloroquine (HCQ) has been identified during decades of trial and error treatments for surfactant dysfunction disorders. Together with promising in vitro data, HCQ has the potential as an innovative lead compound for this group of diseases.

During a previous FP7 project ending 2016 the highest priority for further study in clinical trials was assigned to HCQ due to greatest patients´ needs, wide off-label usage, the results of a worldwide Delphi process and direct recommendation from the Committee for Orphan Medicinal Products (COMP) members. As part of that project the most suitable study design was developed.

This randomized phase 2b double blind clinical study of HCQ in patients with surfactant dysfunction disorders has a design which allows a flexible beginning, either the initiation or the withdrawal of HCQ. The primary objective is to investigate if HCQ improves the oxygenation compared to placebo in a group of 60 well defined subjects with surfactant dysfunction disorders. Secondary objectives of the project will generate important knowledge on patient reported outcomes, health economics and other parameters.

Intervention type: therapeutic medicinal product

This study evaluates the effect of iron chelation as a therapeutic strategy to slow the progression of Parkinson's disease. Half of participants will receive the deferiprone to 15 mg / kg twice daily morning and evening (30mg / kg per day), while the other half will receive a placebo. The treatment lasts nine months.

Population: adult | Intervention type: therapeutic medicinal product

The purpose of this study is to determine if systemic cooling to a target temperature of 34 to 35°C, started within six hours of symptom onset and maintained for 24 hours, improves functional outcome at three months in patients with acute ischaemic stroke.

Population: adult | Intervention type: therapeutic medical device

Antiseptic Silver Alloy-Coated Silicone Urinary Catheters seems to be a promising intervention to reduce urinary tract infections; however, research evidence cannot be extrapolated to spinal cord injured patients.
The study is an open, randomized, multicentric, and parallel clinical trial with blinded assessment. The study includes spinal cord injured patients who require at least seven days of urethral catheterization as a method of bladder voiding. Participants are on-line centrally randomized and allocated to one of the two interventions (Antiseptic Urinary Catheters or Conventional Catheters). Catheters are used for a maximum period of 30 days or removed earlier at the clinician criteria.
The main outcome is the incidence of urinary tract infections by the time of catheter removal or at day 30 after catheterization, the event that occurs first. Intention-to-treat analysis will be performed, as well as a primary analysis of all patients.
The aim of this study is to assess whether silver alloy-coated silicone urinary catheters reduce urinary infections in spinal cord injured patients.

Population: adult | Intervention type: therapeutic medical device

Patients presenting with multiple innumerable liver metastases will probably never come to resection, however, for all others, including patients with numerous multiple metastases or large metastases,resection should be considered after limited chemotherapy.
There is consensus for a backbone chemotherapy consisting of fluoropyrimidine + oxaliplatin. FOLFOX was used in the previous EORTC study and is again recommended.

The addition of targeted agents to standard chemotherapy in the perioperative strategy for mCRC might increase the ORR and R0 resectability, without significant increase in toxicity, therefore translating to a better outcome.
It was therefore decided to design an open label, randomized, multi-center, 3-arm late phase II study.
Arm A: (standard) mFOLFOX6 + Surgery Arm B: (experimental) mFOLFOX6 + Bevacizumab + Surgery Arm C: (experimental) mFOLFOX6 + Panitumumab + Surgery

Population: adult | Intervention type: therapeutic medicinal product

This is a pragmatic randomised controlled trial (PRCT) evaluating the superiority of CT over ICA concerning effectiveness in stable chest pain patients with intermediate pretest probability of coronary artery disease.

Population: adult

The purpose of this study is to evaluate the safety and immunogenicity of a new live attenuated vaccine against whooping-cough. It is a phase1, single centre, dose-escalating, placebo-controlled study on a genetically modified B. pertussis strain given as a single intranasal dose to healthy adult male volunteers.

Effective vaccines are needed to protect young infants (from 0 to 6 months, today the most vulnerable age group), preferably after a single administration very early in life. The successful outcome of this project would constitute an important milestone towards nasal vaccination of infants, possibly at birth with a novel, single-dose pertussis vaccine. Our ultimate aim is to protect infants in the most vulnerable age group, before the regular vaccination schedule using already available vaccines is applied. The ultimate aim is thus not to replace current vaccination schedules with available vaccines, but to add a first nasal vaccination to protect very early in life.

Population: adult | Intervention type: vaccine

A randomised double blind dose non-inferiority trial of a daily dose of 600mg versus 300mg versus 100mg of enteric coated aspirin as a cancer preventive in carriers of a germline pathological mismatch repair gene defect, Lynch Syndrome. Project 3 in the Cancer Prevention Programme (CaPP3).

Population: adult | Intervention type: therapeutic medicinal product

The purpose of this study is to investigate the efficacy of an engineered cartilage transplant (N-TEC) in comparison to a cell-activated matrix (N-CAM) for the treatment of articular cartilage lesions in the knee. The main innovations in this trial are the use of nasal chondrocytes and the implantation of a tissue in contrast to cells seeded on a matrix. The goals of the trial are to: (i) evaluate whether implantation of a more mature graft (tissue therapy) is beneficial for the quality and durability of the repair tissue and the clinical outcome, (ii) determine the potential of the mature graft to integrate with the adjacent cartilage and form hyaline repair tissue and (iii) assess the efficacy of each treatment in correlation to the characteristics of the defect (e.g. acute versus chronic setting).

Population: adult | Intervention type: therapeutic medicinal product

Prospective Multicentric European trial for Cervical cancer, not previously treated, with tumour biopsies, and blood collection for molecular analysis at predetermined time points.

Population: adult