A Multicenter Phase 1 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 Idiopathic Small Fiber Neuropathy (iSFN)
This research is being done to study a possible treatment for refractory pain due to small fiber neuropathy (iSFN). 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 (iSFN).
• Diagnostic characterization of idiopathic Small Fiber Neuropathy (iSFN) without impaired or abnormal glucose metabolism performed according to the Analgesic, Anesthetic, and Addiction Clinical Trial Translations, Innovations, Opportunities and Networks (ACTTION) criteria.
• Medical record documentation that pain is refractory to 2 of 3 categories of first line medical therapy for at ≥ 6 months prior to screening.
• Serum sample negative for pre-existing anti-AAV9 antibodies determined by assay detection limit