Phase 12 Study to Test Safety and Tolerability of Single Dose ST-503 Gene Therapy Delivered into the Spinal Fluid for Treating Refractory Pain from Small Fiber Peripheral Neuropathy

What this Trial Is About

Researchers are evaluating an experimental gene therapy called ST-503 for people who have refractory pain caused by small fiber neuropathy SFN, a condition where small nerve fibers in the skin are damaged. This condition leads to painful tingling or burning sensations, especially in the hands and feet. The study aims to test the safety and tolerability of this gene therapy and to see if it can reduce the refractory pain associated with SFN. The gene therapy works by delivering a modified gene intended to reduce pain signals related to the Nav1.7 protein in nerve tissues. The study involves a randomized, double-blind, sham-controlled design where participants will receive either a single dose of the ST-503 gene therapy delivered intrathecally into the spinal fluid or a sham procedure with no active treatment. The study includes dose escalation to assess safety at different levels. Participants will be monitored closely throughout the study period to evaluate side effects and pain levels. During the 12 weeks following treatment, participants will undergo assessments including pain intensity measurements, sleep interference, anxiety and depression scales, and questionnaires about neuropathy symptoms and disability. Researchers will monitor treatment-emergent adverse events to assess safety and tolerability. The study will also evaluate changes in pain scores and other quality-of-life measures. The total duration of participant involvement covers the treatment and follow-up assessments over 12 weeks.

Safety and Tolerability Study of ST-503 for Refractory Pain Due to Peripheral Neuropathy (Small Fiber Predominant, SFN)