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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT02061137
Date of registration: 27/08/2013
Prospective Registration: Yes
Primary sponsor: University Hospital, Basel, Switzerland
Public title: Study to Assess Safety and Efficacy of Fingolimod in Children With Rett Syndrome FINGORETT
Scientific title: A Phase 1 Clinical Study to Assess Safety and Efficacy of Oral Fingolimod (FTY720) in Children With Rett Syndrome.
Date of first enrolment: August 2013
Target sample size: 6
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT02061137
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 1/Phase 2
Countries of recruitment
Switzerland
Contacts
Name:     Ludwig Kappos, Prof.
Address: 
Telephone:
Email:
Affiliation:  Department of Neurology - University Hospital Basel - Switzerland
Key inclusion & exclusion 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



Age minimum: 6 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Rett's Syndrome
Intervention(s)
Drug: fingolimod (FTY720)
Primary Outcome(s)
Levels of Brain derived neurotrophic factor (BDNF) in blood and cerebrospinal fluid before and under treatment [Time Frame: change of BDNF measured at Baseline, at first dose, at 6 and at 12 months after start of treatment.]
Secondary Outcome(s)
Secondary ID(s)
CFTY720D2201T
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Novartis
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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