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NCT06980948PHASE1, PHASE2Recruiting

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

Sangamo Therapeutics

Start Date

12/4/2025

Completion Date

7/1/2028

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 it 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.

Eligibility Criteria

Age Range: 18 years to No maximum

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 not on a stable dose of opioids; if on a stable dose, the dose may decrease over the course of the study but should not be increased. 2. History of known alcohol abuse, opioid analgesic abuse, or illicit drug abuse within 2 years of Screening. 3. Positive urine test for drugs of abuse (including opiates, benzodiazepines, amphetamines, cocaine, barbiturates, and phencyclidine) without prescription and investigator approval, at Screening and Day -1. 4. Use of cannabinoids is not permitted. 2. Persons with Fabry's disease, with erythromelalgia, with peripheral neuropathies due to alcohol or drug toxicity, or with diagnosed channelopathies 3. Procedure-related: 1. Contraindications to LP, general anesthesia or sedation 2. Any medical disorders that, in the opinion of the Investigator, could interfere with LP including but not limited to evidence for a pressure gradient between supratentorial and infratentorial compartments, Arnold-Chiari malformation, bleeding diathesis, clinically significant coagulopathy, thrombocytopenia, increased intracranial pressure, or spine disease or past surgical procedures involving the spine 4. Infectious disease-related: 1. Active viral infection or bacterial 2. A severe infection (e.g., pneumonia, septicemia, central nervous system infections \[e.g., meningitis, encephalitis\]) within 12 weeks prior to Screening 5. Hepatic disease- and hepatotoxic medication-related: 1. Presence of clinically relevant liver disease 2. Hepatic dysfunction as indicated by one or more of the following: i. Albumin ≤ 3.5 g/dL ii. Total bilirubin \> 1.5 x ULN and direct bilirubin ≥0.5 mg/dL iii. Alkaline phosphatase (ALP) \> 2 x ULN iv. Alanine transaminase (ALT) or aspartate transaminase (AST) \> 1.5 x ULN 3. Hepatotoxic medications should be avoided during the study period including acetaminophen exceeding 4 gm/day unless essential to patient's treatment, approved by investigator, and hepatic dysfunction is not identified 4. Hepatotoxic supplement use during the study period 6. Cancer-related: a. History of cancer, including B-cell cancers, within 5 years of Screening i. Exceptions to this exclusion are fully excised non-melanoma skin cancers, non-metastatic prostate cancer, and fully treated ductal carcinoma in situ of the breast, provided subject has been stable for at least 6 months b. Previous autologous or allogeneic bone marrow transplant, peripheral stem cell transplant or solid organ transplantation 7. Previously received gene or cellular therapy

Interventions

GENETIC

ST-503

PROCEDURE

Sham (No Treatment)

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Conditions

Chronic Neuropathic Pain

Locations

HonorHealth

Scottsdale, Arizona 85260

United States

University of Arkansas for Medical Sciences

Little Rock, Arkansas 72205

United States

The University of California, San Diego

La Jolla, California 92093

United States

Johns Hopkins University

Baltimore, Maryland 21218

United States

Massachusetts General Hospital

Boston, Massachusetts 02114

United States

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire 03766

United States

Columbia University Irving Medical Center (CUIMC) and New York-Presbyterian Hospital (NYPH)

New York, New York 10027

United States

University of North Carolina Medical Center

Chapel Hill, North Carolina 27514

United States

Vanderbilt University

Nashville, Tennessee 37235

United States

University of Utah

Salt Lake City, Utah 84112

United States

Virginia Commonwealth University

Richmond, Virginia 23284

United States