NCT04768166

Testing Miglustat Administration in Subjects With Spastic Paraplegia 11

Phase 2 Pharmacological Trial to Evaluate the Safety of Miglustat Administration in Subjects With Spastic Paraplegia 11 (TreatSPG11)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: IRCCS Fondazione Stella Maris · Academic / Other
• Sex: All
• Age: 14 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. Studies performed in skin cells (fibroblasts) from SPG11 patients, mice and zebrafish models of the disease showed that the material accumulated in the lysosomes is made of glycosphingolipids (GSL). Miglustat is a drug that inhibits an enzyme called glucosylceramide synthetase (GCS) which is used for the production of GSL. Miglustat, therefore, helps to delay the production of GSL. This study aims to collect preliminary data on the safety of miglustat on the SPG11 disease and to assess biomarkers.

Detailed description

We will analyze the safety of Miglustat

Conditions

• Hereditary Spastic Paraparesis

Interventions

Type	Name	Description
DRUG	Miglustat 100 MG	100mg/TID in 4w then 200mg/TID in 8 w

Timeline

• Start date: 2021-06-15
• Primary completion: 2021-08-30
• Completion: 2021-09-15
• First posted: 2021-02-24
• Last updated: 2022-04-11

Locations

1 site across 1 country: Italy