Clinical Trial

Disease: Refractory pain due to small fiber neuropathy (iSFN), (NCT06980948)

Disease info:

Small fiber neuropathy (SFN) is a form of peripheral neuropathy. It occurs when something damages small nerve fibers in the skin, causing symptoms that include painful tingling or burning sensations in the hands and feet. The condition also can affect other small nerve fibers that manage essential body functions, such as blood pressure or heart rate.

SNF symptoms can be severe and affect overall quality of life. While small fiber neuropathy isn’t a serious medical condition, it may be a sign of other conditions that could be life-threatening. Healthcare providers treat the condition by focusing on the underlying causes and with medication that helps with SFN’s painful symptoms.

Symptoms of small fiber neuropathy vary, but common early symptoms are “pins-and-needles” pain or a burning sensation in the hands and feet. These symptoms may come and go or be constant and often worsen over time. Other symptoms include:

  • Feeling lightheaded or fainting.
  • Heart palpitations.
  • Numbness.
  • Not being able to feel pain and temperature in a small spot on your body.
  • Stomach cramps.
  • Sweating more than usual.

About half of small fiber neuropathy cases happen for no known reason. Healthcare providers may call this idiopathic small fiber neuropathy (iSFN). Research shows several medical conditions, inherited disorders or exposure to toxins may cause SFN. These include diabetes, alcohol use disorder, and infectious diseases such as HIV.

Antidepressants, anticonvulsants, opioids and topical therapies are potential treatment options, although no long-lasting or curative therapies are currently available for iSFN patients, leading to a high unmet medical need for this patient population.

Sources: Cleveland Clinic and Sangamo Therapeutics

Frequency:
Based on one study, researchers estimate that 53 out of 100,000 worldwide have small fiber neuropathy. But that estimate may be low: Researchers believe not everyone who has the condition receives a diagnosis.
Official title:
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)
Who:

Name: Patient Advocacy

Phone Number: 510-307-7266

Email: clinicaltrials@sangamo.com

Partners:
Locations:

United States, Arkansas 

Little Rock, Arkansas, United States, 72205

University of Arkansas for Medical Sciences

United States, Maryland 

Baltimore, Maryland, United States, 21218

Johns Hopkins University

United States, Massachusetts 

Boston, Massachusetts, United States, 02114

Massachusetts General Hospital

United States, New Hampshire 

Lebanon, New Hampshire, United States, 03766

Dartmouth Hitchcock Medical Center

United States, New York 

New York City, New York, United States, 10027

Columbia University

United States, Tennessee 

Nashville, Tennessee, United States, 37235

Vanderbilt University

United States, Utah 

Salt Lake City, Utah, United States, 84112

University of Utah

United States, Virginia 

Richmond, Virginia, United States, 23284

Virginia Commonwealth University

 

Study start:
Jul. 1, 2025
Enrollment:
27
Gene editing method:
Zinc finger nuclease repressor
Type of edit:
Transcriptional repression
Gene:
SCN9A
Delivery method:
Adeno-associated virus (AAV) - In-vivo
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Not yet recruiting

Description

This research is being done to study a possible treatment for refractory pain due to small fiber neuropathy (iSFN).

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. About half of small fiber neuropathy cases happen for no known reason. Doctors may call these cases "idiopathic small fiber neuropathy" or "iSFN".

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 iSFN disease. The gene will be delivered into your cells using a special delivery tool called a vector.

Last updated: Jun. 23, 2025
close
Search CRISPR Medicine