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Clinical Trial Summary

Hereditary spastic paraparesis type 11 (SPG11) is caused by mutations in the SPG11 gene that produces spatacsin, a protein involved in lysosomal function.


Clinical Trial Description

Several experiments on subjects affected by neurodegenerative diseases with dysfunction of the autophagic-lysosomal system show that trehalose improves the pathological phenotype. This evidence indicates that trehalose could be used in patients with SPG11 to try to prevent the accumulation of glycosphingolipids at the lysosomal level and induce the genesis of new lysosomes. This study aims to record clinical data of 20 patients with SPG11 who take trehalose during 12 months. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04912609
Study type Observational
Source IRCCS Fondazione Stella Maris
Contact
Status Completed
Phase
Start date June 30, 2021
Completion date July 30, 2022

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