Clinical trials are tightly regulated by laws, which were made to protect participants, while ensuring that the drug in development will not be released for use as a patient treatment until it is safe and effective..

These trials are advanced in steps, each of which has to be successful in order to move on to the next phase - see phases I to IV below:

• Phase I trials determine how safe a compound is in healthy volunteers (looking out for potential undesired side effects).
• Phase II trials determine safety and the optimal dose in patients (the amount of the compound and how often it should be taken).
• Phase III trials determine the compound’s effectiveness in patients.
• Phase IV trials may be performed after the drug is already licensed and usually look for the effect of the drug outside the limited specifications of earlier clinical phase trials.

Each phase trial must show that it was successful in order to move on to the next phase trial.

Due to the complexity of these processes, it typically takes years from early development to marketing of a drug. In this process, many compounds fail to demonstrate effectiveness or for other reasons their development may be stopped.

More information on how drugs are developed may be found in EUPATI- European Patients' Academy

• Clinical trials registration

  By law, clinical trials must be registered before recruiting participants.

  Examples of such registration sites are the US National Institutes of Health (NIH) “clinical trials” or the European “EUDRACT” and these are available to search by professionals and the public.

• Drugs used to treat people with bronchiectasis

  In bronchiectasis, many drugs are used although they were developed for a different use (indication).

  One example is Colistin - an antibiotic that was developed for use as an intravenous antibiotic - but is commonly used for long-term inhalation in bronchiectasis.

  Such use is sometimes referred to as off-label, meaning that it is used differently from the original registration.

Figure 1. Antibiotics (Drugs that are active against bacteria)

* Notes

Inhaled Amikacin (ALIS) - For infection with non-Tuberculous mycobacteria (NTM); marketed (USA) and ongoing
Inhaled Colistin - Marketed in some countries, other phase III studies ongoing
Aztreonam inhalation – Cayston - Completed study did not achieve predefined outcome; phase II to begin
Inhaled dual release Ciprofloxacin – Trials ongoing
Inhaled dry powder Tobramycin – Phase II completed

Figure 2. Drugs that target inflammation

* Notes

Inhaled A1AT – a protease inhibitor - A1AT- alpha 1 antitrypsin

Figure 3. Other drugs

*Notes

Hypertonic saline - Use as physiotherapy adjunct
Inhaled mannitol - Phase III completed
ENaC inhibitor for patients with PCD (VX 371) – CLEAN-PCD - For PCD- Primary ciliary dyskinesia, a specific cause of bronchiectasis

• Figure 1: Antibiotics (Drugs that are active against bacteria)

  
  

There are several ways you could get involved in research and clinical trials - see below - and also Get involved in research (scroll to the bottom of this page)

• Participant in a clinical trial (including pros and cons)

  Taking part in a clinical trial is entirely up to you. Before deciding to participate, a researcher (usually a doctor), will explain how the trial is conducted and what other options are available for you if you decide not to participate.

  Usually, there are very strict rules when conducting a clinical trial that include:

  • The dose (amount) of medication tested.
  • The length of time you will take part in the study.
  • The number and timing of visits to the centre.

  These, and other factors, are predefined in a protocol that has to be followed carefully.

  Many trials test a drug in comparison to a 'placebo' drug: a compound that looks the same, but is not active. Some participants are given an active drug, and others the placebo drug for some or the entire time of the trial. The reason for this is to isolate the effect of the drug from other effects of simply being in a clinical trial that may be beneficial e.g. more frequent visits to the clinic. For this reason, both the participant and the staff at the study centre, do not know which participants are taking an active drug or not - this is called 'blinding'.

  Advantages to being a participant:

  • Having access to treatment that may be effective but is yet unavailable outside of the clinical trial.
  • Clinic visits are more frequent and more emphasis is given to making sure that participants are getting proper care.
  • You will be helping the patient community in the development of new drugs.

  Disadvantages to being a participant:

  • Some new drugs may actually be harmful to a small percentage of people. In advanced study phases, drugs have already been tested and major harm is excluded. However, rare effects may not be evident until a large number of participants have taken the drug.
  • The length of time and travel involved for study visits and taking drugs at home.
  • Some of the trials involve tests that may be inconvenient or associated with a small risk (blood tests, CT scans).
  • Usually, you may participate in only one trial at a time, so participating in a clinical trial usually disqualifies you from participating in other trials.

  How do I get involved?

  • Start by asking your healthcare professional or specialist whether they are aware of any clinical trials relating to your condition and whether the trials are recruiting patients.
  • Contact research organisations (or clinical research organisations, CROs) who are specialised in performing clinical trial studies mainly for pharmaceutical companies and may advertise widely for participants in their trials.
  • Search clinicaltrials.gov or view all trial studies in bronchiectasis.

• Involvement with a local ethics committee

  Ethics committees are there to safeguard the rights, safety, dignity and well-being of research participants independently of research sponsors.

  Usually, they are made up of lay members and healthcare professionals.

  Contact your local hospital if you want more information about being involved with a local ethics committee.

• Involvement in identifying topics for research

  You could consider working with healthcare professionals, a university, health services, patient organisations or a pharmaceutical company involved in developing treatments or diagnostic tools.

  Try to find and join a local patient and public involvement group with links to clinical or academic researchers, or search online for opportunities.

  Read about how patients and healthcare professionals were involved in developing bronchiectasis research priorities.
  

  
  

• Designing medical research

  If you would like to be involved in designing medical research, recruiting patients to take part, or making the results of research available to other patients, the simplest route is to work with a patient organisation.

  Search the European Lung Foundation Patient Organisation Network.

Access the European Patient Ambassador Programme (EPAP) free, online module on 'Supporting research and development' to get more detailed information and knowledge about clinical trials, health research and getting involved.

The module is available in English, Dutch, French, German and Italian.

Register at www.epaponline.eu.