What is a clinical trial?
The I-Motion Institute, by facilitating pediatric trials in rare diseases, is helping to accelerate the research for new treatments, new diagnoses, new clinical assessment tools and daily patient management.
A clinical trial (or therapeutic trial) is a scientific experiment in which a potential treatment is studied in humans in order to evaluate its tolerance and efficacy.
A clinical trial aims to verify in a significant number of people that a new treatment is well tolerated and it is effective in the disease being treated. It is a preliminary step which is inevitable to the provision of a new treatment.
Participate in a clinical trial
Participating in a clinical trial is a personal choice. It means to enter a strictly defined protocol to ensure the safety of the participants, and to obtain a reliable and relevant result. It means to accept exclusive requirements for additional visits and hospital examinations.
The different types of visits
- The selection or screening visit
The investigating physician carries out a complete clinical examination of the patient, reads the entire medical file and verifies that the inclusion and exclusion criteria are respected. If a criterion is not met, the patient is not eligible for study and cannot be included.
- Inclusion visit and randomization
The investigating physician offers the parents to participate for their child in a clinical study if the child answers the selection criteria. He/she explains the information notice and then gives it to the family. On the day of the appointment he/she explains in detail the protocol to them, answers their questions. If the parents agree to participate in the study, the physician then collects their signed and dated consent.
The investigator must then notify the sponsor of the inclusion. Additional examinations will be carried out if necessary. When the promoter has validated the inclusion, if necessary one of the studied treatments (randomization) may be assigned to the patient. The patient’s visits are then scheduled according to the schedule under the protocol.
- Patients’ follow-ups and end of the study visit
The investigator performs a complete clinical examination of the patient at each on-site visit, including all adverse events that have occurred since the last visit and all treatments taken by the patient. The evaluations provided for in the protocol are carried out (motor scales, quality of life questionnaires, blood tests, etc.). The end-of-study visit takes place the same way.