Trehalose Administration in Subjects With Spastic Paraplegia 11 (3AL-SPG11) (3AL-SPG11)
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Hereditary spastic paraparesis type 11 (SPG11) is caused by mutations in the SPG11 gene that produces spatacsin, a protein involved in lysosomal function.
Condition or disease
Hereditary Spastic ParaplegiaSpastic Paraplegia Type 11
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.
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Layout table for eligibility information
Ages Eligible for Study:
10 Years and older (Child, Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Subjects will be enrolled in our centre (IRCCS Fondazione Stella Maris) as part of the primary care during regular visits.
Confirmed diagnosis of SPG11
Written signed informed consent
Diagnosis of other concomitant neurodegenerative diseases
taking other experimental drugs within 30 days of the first Study visit (T0) and during the study