ST-503 for Small Fiber Neuropathy

Recruiting at 10 trial locations

Overseen By Patient Advocacy

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Sangamo Therapeutics

Must not be taking: Cannabinoids, Hepatotoxic medications

Disqualifiers: Alcohol abuse, Drug abuse, Cancer, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

27 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ST-503 for individuals experiencing ongoing pain from small fiber neuropathy (SFN). The treatment delivers a modified gene to reduce pain signals reaching the brain. Participants will receive either the actual treatment or a placebo (a harmless, inactive substance), enabling researchers to compare results. Ideal candidates for this trial have been diagnosed with small fiber neuropathy and have experienced persistent pain despite using standard pain medications for at least six months. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does mention that you should not use cannabinoids and should avoid hepatotoxic medications (those that can harm the liver) during the study. If you are on a stable dose of opioids, you may continue, but the dose should not be increased.

Is there any evidence suggesting that ST-503 is likely to be safe for humans?

Research has shown that ST-503 is being tested for safety and tolerability in treating pain from small fiber neuropathy (SFN). Early studies examine patient reactions to a single dose of ST-503. Although detailed results are not yet available, reaching this stage suggests initial confidence in its safety.

In this trial, participants receive ST-503 to directly target pain signals related to SFN. The goal is to reduce pain by altering specific nerve signals that reach the brain. While specific side effects have not been disclosed, the trial is designed to closely monitor any potential negative effects, with safety as a primary focus. Any serious reactions will be carefully observed.

Overall, while exact safety data is still being gathered, ongoing testing indicates that ST-503 might be well-tolerated in humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about ST-503 for small fiber neuropathy because it offers a novel approach compared to current treatments, which mostly include pain relievers and anti-seizure medications. Unlike these standard options, ST-503 works by targeting the underlying nerve damage specifically linked to small fiber neuropathy, potentially addressing the root cause rather than just managing symptoms. This investigational agent might provide more effective and long-lasting relief, which is a significant advancement for patients suffering from this painful condition.

What evidence suggests that ST-503 might be an effective treatment for small fiber neuropathy?

Research has shown that ST-503, which participants in this trial may receive, targets a specific gene to reduce certain channels in nerve cells that send pain signals. These channels are linked to pain, so reducing them might lessen pain. Animal studies have shown promise in reducing pain signals with this approach. Although human studies remain limited, the treatment aims to help with difficult-to-treat pain from small fiber neuropathy (SFN). Early results suggest that ST-503 could effectively manage this type of pain by blocking pain signals before they reach the brain. ^{1 2 3 5 6}

Who Is on the Research Team?

Medical Monitor

Principal Investigator

Sangamo Therapeutics Inc.

Are You a Good Fit for This Trial?

This trial is for individuals with small fiber neuropathy causing severe pain that hasn't improved after trying at least two types of standard pain treatments for six months. Participants must not have certain health issues like liver disease, recent cancer, or a history of substance abuse and should not be planning surgeries soon.

Inclusion Criteria

Serum sample negative for pre-existing anti-AAV9 antibodies determined by assay detection limit

My pain hasn't improved after trying two different pain relief methods for 6 months.

I have been diagnosed with iSFN without diabetes.

Exclusion Criteria

I have a significant liver condition.

I am scheduled for surgery within three months or during the study.

Any active legal action related to pain disorder

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of intrathecal ST-503 gene therapy to assess safety and tolerability

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with assessments at Weeks 12, 24, and 52

52 weeks

Multiple visits (in-person and virtual)

What Are the Treatments Tested in This Trial?

Interventions

• ST-503

Trial Overview The study tests ST-503, a gene therapy aimed to reduce pain by altering the way nerve signals are sent to the brain. It's compared against no treatment (sham). The goal is to see if ST-503 can effectively decrease chronic neuropathic pain in participants.

How Is the Trial Designed?

Group I: Investigational Agent Experimental Treatment 1 Intervention

Group II: Sham Controlled Study Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Sangamo Therapeutics

Lead Sponsor

Trials

29

Recruited

950+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06980948

Safety and Tolerability Study of ST-503 for Refractory Pain ... This research is being done to study a possible treatment for refractory pain due to small fiber neuropathy (SFN). ST-503 is intended to ...

2. trialx.com trialx.com/clinical-trials/listings/324485/safety-and-tolerability-study-of-st-503-for-refractory-pain-due-to-peripheral-neuropathy-small-fiber-predominant-sfn/

Safety and Tolerability Study of ST-503 for Refractory Pain ... 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 ...

3. sangamo.com sangamo.com/programs/clinical-trials/sfn/

Small Fiber Neuropathy (SFN) By directly targeting the SCN9A gene, ST-503 has shown to selectively reduce the expression of Nav1.7 sodium channels in sensory neurons in animal models and ...

4. synapse.patsnap.com synapse.patsnap.com/article/fda-clears-sangamos-ind-for-st-503-to-treat-idiopathic-small-fiber-neuropathy

FDA Clears Sangamo's IND for ST-503 to Treat Idiopathic ... The Phase 1/2 trial will evaluate the safety and effectiveness of ST-503 for iSFN, a form of peripheral neuropathy that causes severe symptoms ...

5. withpower.com withpower.com/trial/st-503-for-small-fiber-neuropathy-420d9

ST-503 for Small Fiber Neuropathy 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 ...

6. insideprecisionmedicine.com insideprecisionmedicine.com/topics/precision-medicine/fda-clears-sangamos-st-503-for-chronic-neuropathic-pain-clinical-trials/

FDA Clears Sangamo's ST-503 for Chronic Neuropathic ... This study aims to assess the safety, tolerability, and initial efficacy of a one-time intrathecal dose of ST-503. If successful, Sangamo plans ...