An MRI-Study to Characterize Cerebrospinal Fluid Geometry and Dynamics in Individuals with Rett Syndrome

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Updated on 1 June 2022


Knowledge of the CSF geometry and dynamics in RTT will allow for customized intrathecal (IT) drug delivery for subjects with RTT and may help improve gene therapy distribution and exposure at the CNS target areas.
Delivery of drugs to the CSF to help treat central nervous system diseases has shown promising pre-clinical and clinical results. However, at present, the understanding of CSF geometry and physiology in many central nervous system (CNS) diseases is limited. A more complete understanding and quantification of CSF geometry and physiology is needed to optimize drug therapeutic delivery and exposure to the desired brain regions for various diseases including RTT.
The goal of the proposed study is to, for the first time, quantify CSF dynamics and geometry in RTT using advanced engineering analysis of non-invasive dynamic MR imaging. Results will provide information about CSF dynamics in RTT patients for intrathecal (IT) drug delivery, which has potential to improve gene therapy and other therapeutic modalities. 


One MRI imaging session (lasting less than 1 hour) will be conducted. This MRI examination is not part of normal clinical routine for the subjects enrolled in the study. No MRI contrast enhancement agents will be used. However, due to the age of participants, subjects who are unable to remain static during the MRI scan procedure may undergo sedation. Total MR imaging time will be up to 60 minutes per session depending on the age of the subject, size of subject, and heart rate (See Table S2-1).
MRI results will be sent, within 24 hours, to the imaging vendor and Investigator for further evaluation. Any potentially clinically actionable MRI findings will be communicated back to the Investigator by the imaging vendor. Detailed information on MR sequences, parameters, and data analysis will be outlined in the MRI manual.
Anesthesia or sedation will be required to complete this study. The means will be determined by the local study Investigator according to their standard clinical practice for individuals with typical Rett Syndrome.

Condition Rett syndrome
Clinical Study IdentifierTX290604
Last Modified on1 June 2022

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