informations for patients
  • What are clinical trials?

    Many pediatric diseases are still lacking of suitable therapies in terms of safety and efficacy.
    Clinical trials allow us to test whether a treatment works and whether it is safe.
    Clinical trial is defined as any research on human subjects intended to discover or verify the clinical effects of one or more drugs or non-drug related therapeutic procedures (such as surgery, radiation therapy, or medical device) also assessing and studying adverse reactions. In the case of experimental drugs, clinical trials study the mechanism of action, absorption, distribution, metabolism and elimination of the drug to make sure it is suitable for children.

  • Why take part in a clinical trial in pediatrics?

    Children have the right to receive suitable cures, which are effective and safe. The efficacy and safety of a treatment can only be established after quality clinical trials, designed and performed on the population that will use it. More than half of the drugs commonly used in children (and 80% in neonatal age) have only undergone studies in adults, and the results derived have been adapted for their use in pediatrics (the so-called off-label use). Infants, children and adolescents have specific physiological features, which can make the drug ineffective or even toxic if not properly designed for these age groups.

    Children are vulnerable individuals, and their parents or legal guardians must decide their participation in the interest and the wellbeing of their child. It is also important to listen and respect the willingness of the child.
    Families, who decide to involve their child in a trial, contribute to the development of research, have an active role in the health of their child and can have access to cures before everyone and to therapies not commercially available yet.
    One reason that drives parents to enter into a clinical trial is also to help other families who deal with similar situations. Today, our children have a better quality of life and are protected from many diseases once lethal, because of parents in the past have made the decision to allow their children to participate in a clinical trial.

  • Why perform clinical trials in all age groups?

    The child is not a "small adult" but has a body in continuous and progressive evolution.
    A few months infant is different from a teenager.
    Clinical trials also help to identify the most suitable cures for each age group.

  • The pediatric clinical research today

    The overall number of pediatric trials has been increasing since 2007, until of about 350 per year, distributed in various therapeutic areas.

  • When does an investigational drug become available for a pediatric trial?

    Before participating in a clinical trial, several studies have been performed on the investigational drug, i.e. in animals, healthy volunteers and adults.
    Trials are divided into phases; each phase assesses different aspects:

    Phase I: evaluates safety, dosage and possible side effects on a small number of patients (20-80 people). This phase studies very serious diseases or those that have no other treatment options.
    Phase II: phase II trials evaluate how the drug works, its effectiveness and how to improve its safety. This phase includes a greater number of patients (100-300).
    Phase III: involves a large number of patients (1000-3000) to confirm that the drug is effective, and collect all the information on the side effects for its launch on the market.
    Phase IV: this phase is the so-called "post-marketing" it takes place after the launch of the drug on the market in order to monitor its safety.

  • What benefits will your son/daughter receive from participation in a clinical trial?

    Research in pediatrics can only be performed if patients have direct benefits from it. The interest of the patient must always prevail over the interests of science and society. Pain, discomfort, fear and any other expected risk must be minimized, and the patient can be withdrawn from the study at any time and other options on the available therapies will be provided.
    An independent Ethics Committee / Institutional Review Board always assesses the benefits and risks of a trial; the committee is composed of a group of experts, including physicians, statisticians, chemists, experts in bioethics, theologians, nurses.

  • What happens during a clinical trial?

    If the doctor who visits your son/daughter will consider helpful his/her participation in a clinical trial, he will give you an informed consent. This document describes the purpose of the study, its procedures, risks and benefits. The participation of your son/daughter in a clinical trial is bound to the acceptance of the informed consent. You will have time to decide, discuss it with your primary care pediatrician if you want to, and the doctor of the Hospital will answer all of your questions. If you accept, you will be called for visits on a regular basis and the trial team will make sure that your son/daughter takes the medication according to the instructions; the team will check parameters and tests (such as ECG, blood samples, etc.) and it will make sure that there are no side effects.
    You can decide to withdraw your son/daughter from a trial at any time by informing the staff or the doctor. If you wish, your son/daughter will continue to be cured by the Hospital for standard treatments.
    In any case, you are promptly informed about the administered therapies and procedures used during a clinical trial to make you aware and participate in the healthcare of your child.

  • Do you want to know the ongoing trials?

    For the experiments in progress at the Center Trial, see "Clinical studies".

    For the experiments in progress at the Department of Onco-Hematology, contact number 06.6859.3697 and see "Studies cynical" therapeutic area Onco-Hematology.