FOR PATIENTS: clinical trial information

''Despite good results with antibiotics, continuous pulmonary deterioration and death of the CF patient is a reality - Alternative therapeutic strategies are badly needed.''

- IMPACTT  member

Objectives of IMPACTT

The IMPACTT clinical trial will demonstrate the critical preventive and therapeutic effects of this pioneering intervention therapy for the chronic infection of Pseudomonas aeruginosa in CF patients.

The project aims at preventing Pseudomonas aeruginosa infection in CF patient lungs. Thereby, alleviating the negative impact of Pseudomonas on the quality of life of CF patients and their families.

IMPACTT will also explore ways of meaningfully involving patients and family members into clinical CF research.

CFE - As Partner in the IMPACTT Project

Cystic Fibrosis Europe (CFE) is leader of the ‘Dissemination and Exploitation’ work (‘Workpackage 7’) in the IMPACTT project. In this project CFE’s primary goals are:

• CF patient representation
• Spreading knowledge and information about the clinical trial conduct
• To be the contact point for clinical trial participants
• Dissemination and exploitation of project results

In order to promote and improve the involvement of patients and patient's organisations in clinical trials, CFE needs to learn more about the patient’s motivation to join the study and their findings about their participation at the end of the study. This is why CFE developed two questionnaires, which you can find here.

Pseudomonas infection
 

Why is it so important to fight Pseudomonas?

About 50 years ago patients with Cystic Fibrosis died in the first years of their lives. Since then CF specific research, medical treatment and care have been developed, and life expectancy has increased considerably: Many children and adults affected by CF will live into their thirties and forties’, and some of them will even experience life beyond that. But even so Cystic Fibrosis still is a chronic, a debilitating and potentially life threatening medical condition.

Cystic Fibrosis affects many organs, e.g. lungs, pancreas, liver, nasal cavities etc. The IMPACCT project, however, aims at exploring a new way to prevent lung infection by pseudomonas bacteria.

CF patients have a particular risk of Pseudomonas infection

Due to the underlying genetic defect sticky mucus clogs the lungs. This leads to colonization of the lungs with bacteria, chronic inflammation and life-threatening lung infections. Pseudomonas aerigunosa is the most significant bacterial pathogen associated with CF pulmonary disease. Pseudomonas is an opportunistic bacterium which you will find everywhere, but especially in a humid environment, for example sinks. It is very well able to adjust itself to the special conditions in the CF lung and survive there. Therefore CF patients are particularly at risk of Pseudomonas infection due to the viscous mucus lining their airways and compromising their respiratory function.

Current treatment of Pseudomonas infection

Today a pseudomonas infection is treated by repeated courses of antibiotics, either inhaled or as tablets, but mostly intravenously applied which has a negative effect on patients' life quality. This project wants to explore a new way of preventing PA-infection as long as possible.

IgY - a new remedy to prevent Pseudomonas infection?

Research of the past years developed an antibody against Pseudomonas bacteria which is produced from egg yolk. Hens are vaccinated with an alleviated Pseudomonas aerigunosa bacterium. Thus the hens develop antibodies against Pseudomonas which can be found in their eggs also. This antibody is called IgY. By a special procedure for which merely normal water is used the antibody is washed out of the eggs. Thus a medical formulation is obtained. Patients gargle with this formulation. It is expected to form a barrier in their throat and thus prevent that pseudomonas descends from patients’ nasal cavities and throats into the lungs. Past studies with small numbers of patients indicated that IgY might have a potential to form such a “barrier” and showed that the risk for adverse events was rather low.

The European Medicines Agency granted "Anti-pseudomonas IgY" an Orphan Drug Designation in September 2008, and pediatric CF product development was encouraged.

Anti-Pseudomonas IgY

Anti-Pseudomonas IgY is prepared by vaccinating hens with Pseudomonas aeruginosa (PA) bacteria. The hens produce antibodies against PA that are transferred to the egg yolk in high concentration. Anti-Pseudomonas IgY is extracted from the egg yolk. The formulation only contains a water extract from egg yolk without any other additives.

Anti-Pseudomonas IgY Research

The IMPACTT preclinical (IgY research) studies include, the assessment of the:
•Safety studies for the effects of the normal gastrointestinal flora and other potential side effects.
•Anti-Pseudomonas IgY Effect on biofilm formation
•Cross reactivity of IgY against different PA strains
•Pharmacokinetic studies: Duration of the antibodies in the oral cavity
•Identify if there are any antibodies against anti-Pseudomonas IgY (Anti-IgY) produced in the serum of anti-Pseudomonas IgY treated patients
•Effects of the IgY treatment on the inflammatory activity in clinical trial patients (Human efficacy study.).

For more information on the above, please refer to the patients' information.