FAQ about clinical trials
Clinical trials – which are also known as clinical research studies – are medical investigations that are designed to better understand human diseases and medical conditions, with the goal of finding ways to improve their diagnosis, prevention and treatment. Clinical trials are most often conducted to determine if a new treatment (a drug, device, surgical procedure or alternative use of an existing therapy) is safe and effective. Before any new treatment is made available to patients it is mandatory that it be studied in a stepwise series, or “phases” of clinical trials. Each clinical trial phase has a different purpose and answers different questions.
- Phase I: to evaluate safety and identify possible side effects in a small number of either healthy subjects or patients with certain diseases.
- Phase II: to evaluate safety, feasibility and effectiveness, also in a small number of patients.
- Phase III: to confirm effectiveness, monitor side effects, compare treatment with standard or equivalent treatments in a larger number of patients.
- Phase III studies are the primary basis upon which health authorities, such as the US FDA (Food and Drug Administration) and EMA (European Medicines Agency), decide to approve the new treatment and to make it available to patients.
- Phase IV: after a drug is approved and made available to the public, additional studies may be conducted in order to further monitor safety, and/or seek more information about a drug’s risks, benefits, and optimal use in a larger patient population.
Every clinical trial is conducted according to a plan known as a protocol, that is designed to safeguard the participant’s health and rights. The protocol describes the specific criteria that a person must meet in order to be eligible for the trial (also known as the inclusion and exclusion criteria).
In order to determine whether or not a new treatment is successful, it often must be shown to offer additional benefit to treatments that are already available (known as the standard of care). Therefore, participants in trials will often be divided into 2 groups. One group receives the treatment/therapy often in addition to the standard of care and the other receives a placebo (an inactive product that resembles the test treatment, but without its treatment value) in addition to standard of care. The second group is known as the control group.
Randomization is the process by which participants are assigned to the groups. This process avoids any bias with investigators assigning participants to one group or another.
Randomized trials are often conducted during Phase III, and occasionally during Phase II depending upon the disease being studied.
To avoid any bias in observations and to allow scientifically accurate conclusions, the participants (patient and/or members of the research team including the Physician) of the trial do not know which medicine is being used. In some trials, only the patient is not told what is being administered (single-blind study), and in other trials members of the research team are also not told which patients are receiving which medication (double-blinded).
Every participant that decides to enrol in a trial must sign an informed consent document. This document includes such details about the study as its purpose, the duration, the required procedures and who to contact during the trial. The informed consent document also explains risks and potential benefits of participating in the study. After signing the informed consent, the participant will have to attend screening visits, to undergo a number of tests and to answer questions on his/her condition and in order for the research team to collect the medical history of the subject/patient in order to determine if he/she is eligible for the trial. If the person is eligible and decides to participate, he/she will receive the treatment according to the trial protocol and attend hospital/clinic for follow-up tests at defined times over the duration of the trial.
Participating in a clinical trial involves benefits and risks . The specific risks associated with a research protocol are described in detail in the informed consent document and explained by a member of the research team.
Risks to participating in clinical trials include the following:
- Unpleasant, serious, or even life-threatening side effects to experimental treatment.
- Not receiving the experimental treatment if placed in the control group.
- Increased frequency of visits, more tests, longer hospital stays.
Potential benefits :
- Gain access to new research treatments before they are widely available
- Receive regular and careful medical attention
- Help others by contributing to medical research
If you are interested in enrolling in a trial, you should discuss it with your doctor who will then refer you to the Principal Investigator (PI) of the trial. The PI is a doctor who will decide if you are suitable for the trial by meeting all of the eligibility criteria to enrol into the clinical trial.
Celyad is unable to respond to enquiries about participation in its clinical trial. Patients should discuss their medical condition and treatment options with their doctor that will put them in touch with the trial investigators (doctors in charge of the clinical trial sites).