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

NCT02061137 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2018-06-15

No results posted yet for this study

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.

Conditions

  • Rett's Syndrome

Interventions

DRUG

fingolimod (FTY720)

0.5 or 0.25 mg fingolimod orally daily for each of 6 patients with rett syndrome for 12 months

Sponsors & Collaborators

  • Novartis

    collaborator INDUSTRY

  • University Hospital, Basel, Switzerland

    lead OTHER

Principal Investigators

  • Ludwig Kappos, Prof. · Department of Neurology - University Hospital Basel - Switzerland

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-08-31
Primary Completion
2018-04-30
Completion
2018-04-30

Countries

  • Switzerland

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02061137 on ClinicalTrials.gov