Clinical trials
Where can I get information?
- Contact your neurologist who is familiar with the ongoing tests relevant to your case.
- Sign up at MDA Hellas – Association for the Care of Neuromuscular Diseases – to get information on research topics.
- Visit relevant scientific sites such as clinicaltrials.gov and clinicaltrialsregister.eu
What is a clinical trial?
It is a method to study experimental drugs or devices in humans. A strict protocol is followed to preserve participants health while answering research questions. The aim is to measure safety and efficacy of drugs / devices or other therapies.
Who can participate in a clinical trial?
Each trial has specific patient inclusion and exclusion criteria, including fields such as age, sex, type and stage of illness, and treatment history. These ensure the reliability of the trial and patient safety.
Which phases are included?
Prior to exporting any safety and efficacy conclusion, each trial is going through four phases:
Phase I: tests are performed on a small group of individuals for safety assessment, clarification of appropriate dosages and side effects.
Phase II: Tests are performed on larger patient groups to assess efficacy and safety. The choice is made randomly and blindly, and either the drug or the inactive but safe substance, called placebo, is administered.
Phase III: Provides information that leads to a detailed understanding of efficacy, benefits, side effects in large patient groups (hundreds or even thousands) in different centers and lasts several years.
Phase IV: Assesses long-term risks and benefits of treatment as it is now commercially available.
What happens during a clinical trial?
Each clinical trial has different requirements from the participants. In general, however, everyone receives a drug dose for a long time. This may be the active substance or placebo. Everyone is monitored by a multidisciplinary team consisting of doctors of various specialties, nurses and other scientists, who carry out the necessary tests and at the same time appreciate the overall situation of the participants.
What are the benefits of participating?
- Access to medicines before they become widely available – of course, there is no guarantee of the outcome.
- Regular monitoring by qualified personnel.
- Participate in a process aimed at treating serious illnesses and contribute to current medical developments.
What are the disadvantages of participating in a trial?
- Many experimental drugs prove to be ineffective or less effective than everything that is already available commercially. Even when their efficacy is proven, one may not benefit from taking a small dose or placebo.
- It may be necessary to stop taking other medicines in order to be able to participate in the trial.
- There may be side effects, which can be very unpleasant or even dangerous. Of course, strict control and recording of all these data is made and the treatment by the relevant staff is immediate.
- Frequent visits at a hospital or any other medical center involved in clinical trials are often necessary, which can often cause practical problems.
- Some exams or procedures may be painful eg. biopsies.
In any case, detailed information is provided by qualified personnel before the start of the entry.
What does the briefing and the informed consent form include?
A detailed presentation of the important points of the clinical trial is made before anyone decides whether to participate or not. Qualified staff should explain the purpose, duration, procedures, risks, and benefits. All participants sign the necessary consent documents so that they formally ensure they have received the relevant trial information. In case of minors, the document in a comprehensible form is signed by guardians. It is not a contract and the participants can leave the trial whenever they think it is necessary. In such a case, of course, their decision and their reason for leaving the staff must be notified.
Should I participate in a clinical trial?
The participation experience can bring significant results but the decision to participate is personal and a very serious issue. It requires gathering all the necessary information and understanding, as well as answers to the following questions:
- What is the purpose of the trial?
- Why do researchers think it will be effective?
- Has this trial been repeated in the past?
- Who is the sponsor?
- Who has checked and approved the trial?
- What are the credentials of the participating staff?
- Which examinations and procedures must be carried out? Where are they taking place and how often?
- What are the short-term and long-term risks, side effects and benefits? How are they compared to existing drugs?
- How are the results and security of the participants controlled?
- Will I be compensated for any costs, for example moving?
- What the duration of the trial will be?
- When will the test results be available? Will the treatment / device be available after the end of the test?
- Will there be follow up after the test?
More details can be found here: https://drive.google.com/file/d/1InLiKyBY00PMu55KWXLH1-zCXXS_QmIb/view?usp=sharing
Reference
Clinical Trials, Muscular Dystrophy News, 2015, https://musculardystrophynews.com/clinical-trials-101/