After 15 years of research, including three dedicated to the collection of non-profit funding, the MirtaRett project coordinated by Enrico Tongiorgi (UniTS Department of Life Sciences), finally enters clinical practice with the administration of the first therapy to a young patient at the Gaslini Hospital in Genoa.

In February 2025, the Italian Pharmaceuticals Agency (AIFA) received the positive opinion of the National Ethics Committee for Paediatric Studies and thus gave the green light to the first worldwide trial on Mirtazapine in Rett Syndrome, a serious neurological disease that affects almost exclusively girls.

The clinical study, coordinated by the University of Trieste, will initially extend to 15 young patients and will take place in the main Italian reference hospitals for Rett Syndrome. The trial is fully covered by non-profit funds.

The project is supported by the unconditional contribution of Angelini Pharma SpA, Fondazione Canali Onlus, Fondazione Ico Falck and Fondazione Amadei and Setti. In addition to the pharmacological experimentation, the project also provides for the continuous monitoring of patients' vital parameters, such as breathing, heart rate and blood oxygenation. For this purpose, smart T-shirts are used, already distributed to hospitals in Genoa, Siena, Messina and Milan. Originally developed for monitoring athletes, these T-shirts are made of cotton woven with nanofibres capable of detecting the weak electrical signals of the human body and are tailor-made for each patient by the Italian company AccYouRate Group.

What is Mirtazapine?

Mirtazapine is a commercially available drug, therefore more easily accessible and sustainable. To facilitate its intake, a European company has been identified that can produce it in liquid formulation, a solution that is not widespread since the drug is normally marketed globally as tablets. ‘Our laboratory at UniTS Department of Life Sciences was the first in the world to demonstrate that mirtazapine, despite being an antidepressant, acts on broader mechanisms and can improve breathing, motor control, sleep quality and social communication in patients with Rett’s syndrome,’ explains Prof. Tongiorgi.

Access to the trial is open to new patients

In Friuli Venezia Giulia it is estimated that there are three or four girls affected by the syndrome who are not currently included in the study, but the prospects remain encouraging. ‘We hope that the health facilities in the region will also be able to participate in the trial,’ Tongiorgi said.

To ensure the scientific validity of the study, it is necessary to reach a total of 54 patients aged between 5 and 40 years, divided into the ranges 5-10, 11-17 and 18-40 years. Approximately one third have been recruited so the search for new participants is still open.