Study to Assess Safety and Efficacy of Fingolimod in Children With Rett Syndrome

• Status: Completed
• Trial end date: 2018-04-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University Hospital, Basel, Switzerland
• Collaborator: Novartis
• Treatments: Fingolimod Hydrochloride

Criteria

Inclusion Criteria:

• Children fulfilling diagnostic criteria (2001, Hagberg B et al. Eur. J. Paediatr. Neurol. 2002) of Rett Syndrome

• Stages II -IV Hagberg/ Witt-Engerström (Hagberg B, Witt-Engerström I. Am J Med Genet 1986, Hagberg B. Ment Retard Dev Disabil Res Rev 2002)

• Patients older than 6 years old (have had their 6th birthday)

• Written informed consent of parents/ of legal guardian

• Negative testing for pregnancy

• Positive confirmation of a MECP2 mutation

Exclusion Criteria:

• Any uncertainty about diagnosis of Rett Syndrome

• Patients younger than 6 years old (have not yet had their 6thbirthday)

• Additional associated neurological diseases such as a brain malformation

• Patient <15kg body weight at timepoint of screening

• Patients with negative varicella-zoster virus immunoglobulin G (IgG) antibodies

• Pregnancy or breastfeeding for girls in childbearing potential age