Actively Recruiting
A Phase 1 / 2 Double-blind, Randomized, Sham-controlled Study to Evaluate Safety and Tolerability of Single Dose Intrathecal ST-503 Gene Therapy for Refractory Pain Due to Small Fiber Neuropathy
Led by Sangamo Therapeutics · Updated on 2026-06-01
27
Participants Needed
11
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
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.
CONDITIONS
Brief Title
Safety and Tolerability Study of ST-503 for Refractory Pain Due to Peripheral Neuropathy (Small Fiber Predominant, SFN)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Small Fiber Neuropathy (SFN) based on ACTTION criteria
- Pain refractory to at least two of three categories of first-line medical therapy for 6 months or longer
- Negative serum test for pre-existing anti-AAV9 antibodies
You will not qualify if you...
- Using opioid pain medications for less than 3 months or not on a stable opioid dose
- History of alcohol, opioid, or illicit drug abuse within 2 years before screening
- Positive urine drug test without prescription or investigator approval
- Use of cannabinoids is not allowed
- Having Fabry's disease, erythromelalgia, alcohol or drug toxicity-related neuropathies, or diagnosed channelopathies
- Contraindications to lumbar puncture, general anesthesia, or sedation
- Medical conditions that may interfere with lumbar puncture including certain brain or spine disorders
- Active viral or bacterial infections
- Severe infections within 12 weeks before screening
- Significant liver disease or liver dysfunction based on lab values
- Use of hepatotoxic medications or supplements during the study
- History of cancer within 5 years except certain stable non-melanoma skin cancers, non-metastatic prostate cancer, or treated ductal carcinoma in situ
- Previous bone marrow, stem cell, or solid organ transplant
- Prior gene or cellular therapy received
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 12 weeks
Participants receive a single dose of intrathecal ST-503 gene therapy or a sham procedure to evaluate safety and tolerability for refractory pain due to small fiber neuropathy.
Multiple visits over 12 weeks to monitor safety and pain levels
Trial Site Locations
Total: 11 locations
1
HonorHealth
Scottsdale, Arizona, United States, 85260
Actively Recruiting
2
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72205
Actively Recruiting
3
The University of California, San Diego
La Jolla, California, United States, 92093
Not Yet Recruiting
4
Johns Hopkins University
Baltimore, Maryland, United States, 21218
Actively Recruiting
5
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
6
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, United States, 03766
Not Yet Recruiting
7
Columbia University Irving Medical Center (CUIMC) and New York-Presbyterian Hospital (NYPH)
New York, New York, United States, 10027
Actively Recruiting
8
University of North Carolina Medical Center
Chapel Hill, North Carolina, United States, 27514
Not Yet Recruiting
9
Vanderbilt University
Nashville, Tennessee, United States, 37235
Not Yet Recruiting
10
University of Utah
Salt Lake City, Utah, United States, 84112
Not Yet Recruiting
11
Virginia Commonwealth University
Richmond, Virginia, United States, 23284
Actively Recruiting
Research Team
P
Patient Advocacy
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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