NCT05606614
An experimental gene therapy being evaluated in an open-label study, with no promise of benefit.
Ongoing · not recruitingPhase 3
United States*6 – < 22 years
Source updated · Jul 7, 2026
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This is a partial selection. Statuses come from dated sources, not confirmation of an available place. Documented countries and sites are not a complete worldwide list.
6 study/studies in this selection
NCT05898620
Another gene approach, designed to control MECP2 expression.
Ongoing · not recruitingPhase 3
United States*3 + years
Source updated · Jun 8, 2026
Understand this trialNCT07503444
Comparing fenfluramine with placebo in Rett syndrome.
Listed as recruitingPhase 3
Spain*5 – 35 years
Source updated · Aug 17, 2026
Understand this trialNCT07430046
An Italian open-label study of safety, symptoms and daily life.
Recruitment to confirmPhase 2
Italy*5 – < 40 years
Source updated · Feb 24, 2026
Understand this trialNCT04181723
A finished trial with results published in 2023.
Study completedPhase 3
United States*5 – 20 years
Consulted · Sep 8, 2026
Understand this trialNCT04988867
A study in young girls, with a paper published in 2025.
Study ended earlyPhase 2/3
United States*2 – < 5 years
Consulted · Sep 8, 2026
Understand this trial