Clinical Research

A clinical trial is a type of study that investigates the effect of a medicine or treatment in children and adolescents. In a clinical trial, two treatments or drugs are compared, the one to be studied and another already known treatment or an inactive substance, known as a placebo. Neither the parents, nor the children, nor the researcher knows which treatment the patient receives, in order to avoid distorted results. An anonymous lottery is usually used to assign the treatment to the patient.

Regulatory agencies linked to drug authorisations, such as the Food and Drug Administration (FDA) and the European Medicines Agency (EMA), determine the design of clinical trials and, therefore, the use or non-use of placebo. In oncology or ultra-rare disease studies, it is common for placebo not to be used and for the study to be conducted directly on the drug.

At SJD Barcelona Children's Hospital, clinical trials are reviewed and approved by the Fundació Sant Joan de Déu's Drug Research Ethics Committee and the Agencia Española de Medicamentos (AEMPS).

Because children and adolescents are not small adults. Depending on the stage of development, it may be necessary to (1) adjust dosages and (2) have paediatric formulations (syrups, tablets, etc.) available.

Pediatric clinical trials are important because they help researchers and doctors to:

• Find treatments to cure or delay diseases that only occur in childhood and adolescence, to cure or delay diseases that also occur in adults but manifest differently in children and to help improve the quality of life of sick children.
• Finding the right dose of a medicine that causes the minimum number of side effects.
• Develop formulations that children can easily take (syrups, chewing gum, etc.).
• Verify that treatments that are safe in adults are also safe in children.

All children can participate in a clinical trial at our centre, whether they are our patients or not. They have to meet the inclusion criteria of the clinical trial: for example, being of a certain age, having followed a previous treatment, having specific results in a blood test, having a certain disease or even being healthy. These criteria are thoroughly checked by our medical team.

Participation in a clinical trial is always voluntary and altruistic.

Informed consent is a document that contains all necessary information about the clinical trial. Parents/legal guardians of a minor must sign it to acknowledge that they have received the information and that they agree to the child's participation in the trial.

If the trial participant is between 12 and 17 years of age, they must also agree to participate by signing another document called an informed consent form.

For children under 12 years of age, although assent is not legally required, the trial is explained to them with the help of the team and with the participation of their parents/legal guardians.

• Withdraw consent to the study and leave it at any time, while maintaining guaranteed medical care for the child or adolescent.
• Commitment that the confidentiality of the data will be preserved and that no one outside the study will have access to them (you have the right to access, rectify, revoke or cancel the data).
• To be informed about the treatment options available for the patient's disease.
• Receive appropriate and specialised medical care while patients are participating in the clinical trial.
• Not pay for the investigational medicine.
• Ask questions at any time during the clinical trial and about any aspect that impacts on their quality of life (financial impact, hours in hospital, inconvenient travel, number of procedures, etc.).
• Be informed about the progress of the clinical trial.