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)
Sangamo Therapeutics
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).
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-huma…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria 1. Diagnostic characterization of Small Fiber Neuropathy (SFN) according to the Analgesic, Anesthetic, and Addiction Clinical Trial Translations, Innovations, Opportunities and Networks (ACTTION) criteria. 2. Medical record documentation that pain is refractory to 2 of 3 categories of first line medical therapy for at ≥ 6 months prior to screening. 3. Serum sample negative for pre-existing anti-AAV9 antibodies determined by assay detection limit Exclusion Criteria 1. Drug- and alcohol-related: 1. Persons using opioid analgesics for under 3 months or persons who are no…
Interventions
- GeneticST-503
Genomic Medicine
- ProcedureSham (No Treatment)
Sham Procedure
Locations (11)
- HonorHealthScottsdale, Arizona
- University of Arkansas for Medical SciencesLittle Rock, Arkansas
- The University of California, San DiegoLa Jolla, California
- Johns Hopkins UniversityBaltimore, Maryland
- Massachusetts General HospitalBoston, Massachusetts
- Dartmouth Hitchcock Medical CenterLebanon, New Hampshire