NCT06980948

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

A Multicenter Phase 1 / 2 Double-blind, Randomized, Sham-controlled Dose Escalation Study to Determine Safety and Tolerability of Single Dose Intrathecal ST-503 Gene Therapy for Refractory Pain Due to Peripheral Neuropathy (Small Fiber Predominant, SFN)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 27 (estimated)
• Sponsor: Sangamo Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This research is being done to study a possible treatment for refractory pain due to small fiber neuropathy (SFN). ST-503 is intended to deliver a modified copy of the gene which will ideally repress Nav1.7 tissue-related pain signals reaching the brain, which should reduce the refractory pain due to small fiber neuropathy (SFN).

Detailed description

This research is being done to study a possible treatment for refractory pain due to small fiber neuropathy (SFN). Small fiber neuropathy happens when something damages small nerve fibers in your skin, causing symptoms like painful tingling or burning sensations in your hands and feet. Pain originating in the nerves outside of the brain and spinal cord is defined by doctors as neuropathic pain. Scientists have discovered that certain proteins in our bodies called sodium channels are important for communicating pain signals in nerves, specifically, Nav1.7, Nav1.8 and Nav1.9. This first-in-human study will test the use of a type of experimental treatment called "gene therapy." The primary goal is to determine if is safe and well tolerated. The second goal is to determine if it reduces the level of refractory pain due to SFN disease. The gene will be delivered into your cells using a special delivery tool called a vector.

Conditions

• Chronic Neuropathic Pain

Interventions

Type	Name	Description
GENETIC	ST-503	Genomic Medicine
PROCEDURE	Sham (No Treatment)	Sham Procedure

Timeline

• Start date: 2025-10-01
• Primary completion: 2027-07-01
• Completion: 2028-07-01
• First posted: 2025-05-20
• Last updated: 2025-10-28

Locations

10 sites across 1 country: United States

Regulatory

• FDA-regulated drug study
• FDA-regulated device study