PARTICIPATE IN CLINICAL RESEARCH

Clinical research refers to all research carried out on humans (healthy or sick subjects). This term covers studies aimed at advancing knowledge of the mechanisms of the disease (the abnormalities at the root of its development or its worsening: pathophysiological studies), the development of new treatments (therapeutic trials) and diagnostic methods to ensure better patient care.

Clinical research may also be designed to provide a better description of the environmental phenomena that promote the onset of the disease or influence its progression. With this in mind, follow-up studies of cohorts of patients are being set up in order to gain a better understanding of the variability of ALS, which is no longer considered as a homogeneous disease obeying a single determinism, but as a syndrome (i.e. a combination of symptoms), whose contributing factors need to be characterised separately to enable targeted treatments.

This is also known as translational research, the aim of which is to validate in humans, concepts or pathophysiological mechanisms first studied using animal models (mice carrying a genetic anomaly responsible for the disease, for example). 

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When testing the effect of a new drug on humans, it is essential to follow a rigorous scientific protocol that guarantees reliable results. As part of the development of new drugs, clinical trials will comprise four phases:

• Phase 1: first administration in humans, carried out on a small group of healthy or sick volunteers, depending on the drug being evaluated. The aim is to study the tolerance to the product, its fate in the body and its effects on the body.
• Phase 2: first administration, usually in patients. This phase assesses the dosage of the treatment and the safety and efficacy of the drug in small groups of patients. A phase 3 study will only be considered if there is an acceptable level of tolerance by patients.
• Phase 3: therapeutic efficacy study, the aim of which is to demonstrate the effectiveness of the drug on a larger number of patients. The study treatment is compared either to the reference treatment or to a placebo (with no pharmacological effect). This is the last phase, if the drug proves to be effective, before it is marketed as a medicine (which requires marketing authorisation - MA).
• Phase 4: post-marketing surveillance, during which side effects are recorded and analysed to assess patient safety.

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It is important to know that before taking part in a study, the patient (whether sick or healthy) has been informed by the doctor of its objectives, benefits, constraints and possible risks, the timetable for the study and the possibility of withdrawing from the study during the monitoring. All this information is set out in an information memo, and a consent form must be signed before any research-related procedure is carried out.

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