Trehalose Administration in Subjects With Spastic Paraplegia 11 (3AL-SPG11)
- Conditions
- Spastic Paraplegia Type 11Hereditary Spastic Paraplegia
- Registration Number
- NCT04912609
- Lead Sponsor
- IRCCS Fondazione Stella Maris
- Brief 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 13
- 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
- Refusal to sign informed consent
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Changes from baseline in Spastic Paraplegia Rating Scale (SPRS) at 6 and 12 months At baseline, month 6, month 12 Assess changes in score of the Spastic Paraplegia Rating Scale (SPRS) over ± 10%
- Secondary Outcome Measures
Name Time Method Changes in glycosphingolipids and gangliosides plasmatic levels At baseline, month 6, month 12 Assess changes in glycosphingolipids and gangliosides plasmatic levels over ± 10%
Trial Locations
- Locations (1)
IRCCS Fondazione Stella Maris
🇮🇹Pisa, PI, Italy