Clinical studies

Through the well-structured collection of clinical data and the analysis of biological samples, observational studies provide useful information for the knowledge of the disease, which can be used to evaluate the patient in routine visits, to give patients and their families more detailed information about diagnosis and treatment path. In addition, these studies provide valuable information for defining the protocols of interventional studies (trial readiness).

Clinical trials are conducted to assess the safety, tolerability and efficacy of a given treatment in humans for a specific disease.

These studies may involve different therapeutic approaches. Classical pharmacological trials mainly evaluate the action of small chemical molecules, while other innovative pharmacological approaches use biological molecules customized according to the genetic characteristics of patients. These include, for example, gene therapy testing, the use of oligonucleotides (DNA and RNA components encoding proteins) or recombinant proteins such as peptides, enzymes or monoclonal antibodies.

Participation in a clinical trial is very challenging for patients and their families, and, at the clinical level, requires careful monitoring of the patient, the involvement of different professionals and a long follow-up to assess safety and effectiveness in the long term. The inclusion of the most suitable patients in a clinical trial comes primarily from the criteria indicated in the specific study protocol. Even if a patient corresponds to these criteria, however, the inclusion cannot disregard the careful consideration of his clinical and family conditions and a path of sharing and honesty between doctor and patient (and with parents, in the case of paediatric patients), to clarify expectations and balance the benefits and risks of participation in the clinical trial.