Study to Assess Safety and Efficacy of Fingolimod in Children With Rett Syndrome
Status:
Completed
Trial end date:
2018-04-01
Target enrollment:
Participant gender:
Summary
The Trial Objective is to assess safety and efficacy of oral fingolimod (FTY720) in children
older than 6 years with Rett Syndrome. So far there is no established treatment for children
with Rett Syndrome. Therefore a positive result in terms of safety and first indications of
efficacy would path the way to a phase II clinical study with more patients to further test
the hypothesis that fingolimod treatment may slow down the regression of motor and language
skills.