NCT04912609

Trehalose Administration in Subjects With Spastic Paraplegia 11 (3AL-SPG11)

An Observational Study in Subjects With Spastic Paraplegia Type 11 Taking Trehalose

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 13 (actual)
• Sponsor: IRCCS Fondazione Stella Maris · Academic / Other
• Sex: All
• Age: 10 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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.

Conditions

• Hereditary Spastic Paraplegia
• Spastic Paraplegia Type 11

Timeline

• Start date: 2021-06-30
• Primary completion: 2022-04-30
• Completion: 2022-07-30
• First posted: 2021-06-03
• Last updated: 2022-08-03

Locations

1 site across 1 country: Italy