by InTrieste
Italian researchers have received regulatory approval to launch the world’s first clinical trial of Mirtazapine for Rett syndrome, a rare genetic disorder. The trial, known as MirtaRett, will be coordinated by the University of Trieste and conducted at leading Italian medical institutions.
The study, backed by the Italian Medicines Agency (AIFA), will begin in two months and enroll 54 female patients aged 5 to 40 years. It aims to evaluate Mirtazapine’s impact on motor function, communication abilities, sleep, and psychiatric symptoms.
Rett syndrome, caused by MECP2 gene mutations, primarily affects girls, leading to severe developmental regression, loss of speech, and motor impairments. While there is no cure, preliminary studies suggest that Mirtazapine may offer symptom relief.
“This is a major step forward,” said Professor Enrico Tongiorgi, the study’s principal investigator. A retrospective study in Siena previously showed benefits in anxiety, irritability, hand use, and overall disease progression. The new trial will determine if these effects extend to younger patients.
The trial will take place at centers in Milan, Genoa, Siena, and Messina, using an innovative Italian-designed smart T-shirt to track breathing irregularities. Funding comes entirely from nonprofit grants, including Angelini Pharma and several charitable foundations.
“If results are positive, this could become a new standard of care,” Tongiorgi said. Families affected by Rett syndrome are watching closely, hopeful that this research could bring meaningful improvements to patients’ lives.
