Engensis for Painful Diabetic Peripheral Neuropathy

(REGAiN-1B Trial)

Overseen By Louise A. Taber MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Helixmith Co., Ltd.

Disqualifiers: None

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 6 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the long-term effects and safety of a treatment called Engensis for individuals with painful diabetic peripheral neuropathy, a condition causing nerve pain due to diabetes. The researchers aim to assess the treatment's effectiveness over time and its safety when administered through muscle injections. Participants will receive either Engensis or a placebo (a non-active treatment) and must have completed a previous related study. This trial may suit those with ongoing nerve pain from diabetes who participated in the earlier study. As a Phase 3 trial, it represents the final step before FDA approval, offering participants a chance to contribute to potentially bringing a new treatment to market.

Do I need to stop my current medications for this trial?

The trial information does not specify whether you need to stop taking your current medications.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that Engensis is generally safe for people. In previous studies, individuals with painful diabetic nerve damage who used Engensis experienced a noticeable decrease in pain. Importantly, these studies did not find any major safety issues. Another study examined the safety and tolerability of Engensis when injected into leg muscles. This study also supported the idea that Engensis is safe, with participants handling it well. While these results are encouraging, consulting a healthcare provider before joining a clinical trial is always advisable. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for diabetic peripheral neuropathy?

Engensis is unique because it uses a gene therapy approach to address painful diabetic peripheral neuropathy. Unlike standard treatments, such as pain relievers or anticonvulsants that only manage symptoms, Engensis works by delivering a DNA plasmid to stimulate the production of a protein that promotes nerve regeneration and repair. Researchers are excited because this could offer a more effective and long-term solution, potentially reversing nerve damage rather than just alleviating pain.

What evidence suggests that Engensis might be an effective treatment for painful diabetic peripheral neuropathy?

Research shows that Engensis, also known as VM202, may help reduce pain for people with painful diabetic nerve damage. Previous studies have found Engensis to be safe and effective for this condition. In one study, patients who received Engensis reported significantly less pain compared to those who did not. In this trial, participants will receive either Engensis or a placebo. Engensis is a type of gene therapy that repairs nerve damage and encourages new blood vessel growth. While more research is needed, early results are promising for those suffering from diabetic nerve pain. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

Inclusion Criteria

Completed study VMDN-003-2 and consent to enroll in study VMDN-003-2b

Completed study VMDN-003-2 and consent to enroll in study VMDN-003-2b

Exclusion Criteria

There are no specific exclusion criteria for this study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants received intramuscular administration of Engensis or Placebo in the double-blind, randomized, placebo-controlled Phase 3 Study VMDN-003-2

6 months

Extension

This 6-month extension study evaluates the durability of efficacy and long-term safety of Engensis or Placebo without administering any treatment

6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments of pain scores and adverse events

6 months

What Are the Treatments Tested in This Trial?

Interventions

• Engensis
• Placebo

How Is the Trial Designed?

Group I: Engensis Experimental Treatment 1 Intervention

Patients who have received Engensis in protocol VMDN-003-2

Group II: Placebo Placebo Group 1 Intervention

Patients who have received Placebo in protocol VMDN-003-2

Who Is Running the Clinical Trial?

Helixmith Co., Ltd.

Lead Sponsor

Trials

20

Recruited

1,300+

Published Research Related to This Trial

The meta-analysis of 8 studies involving 656 patients showed that Tongmai Jiangtang capsules (TJC) combined with conventional treatment significantly improved nerve conduction velocities in diabetic peripheral neuropathy (DPN) patients compared to conventional treatment alone.

No adverse drug reactions were reported with the use of TJC, indicating a favorable safety profile, but the authors caution that the results should be interpreted carefully due to significant variability in the data across studies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of Tongmai Jiangtang capsule combined with conventional therapy in the treatment of diabetic peripheral neuropathy: a systematic review and meta-analysis. Sun, LX., Li, YY., Xie, YM.[2023]

In a systematic review of 21 studies involving 2425 patients with painful diabetic neuropathy, the average placebo response showed a significant reduction in pain intensity, with a moderate effect size of 0.72, indicating that many patients experienced relief even without active treatment.

The study found that over half (53.3%) of patients in the placebo groups reported adverse events, with 5.1% dropping out due to these events, highlighting the importance of understanding both the benefits and potential risks associated with placebo treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Placebo and nocebo responses in painful diabetic neuropathy: systematic review and meta-analysis. Frisaldi, E., Vollert, J., Al Sultani, H., et al.[2023]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04469270

Study to Assess Safety and Efficacy of Engensis in Painful ... The purpose of this study is to evaluate the efficacy and safety of intramuscular administration of Engensis on pain in participants with painful diabetic ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8212761/

Gene therapy for diabetic peripheral neuropathy VM202 (Engensis), a nonviral plasmid DNA product, is the first gene medicine to enter advanced clinical trials for treatment of painful DPN. VM202 is ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT01475786

NCT01475786 | Safety and Efficacy Study for the ... The purpose of this study is to determine if Engensis (VM202) is safe and effective in treating painful diabetic neuropathy.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10949339/

Update on Treating Painful Diabetic Peripheral Neuropathy At 6 months, 59% of low-frequency SCS plus CMM versus 7% of CMM alone recipients achieved treatment success, which was defined as ≥50% pain ...

5. withpower.com withpower.com/trial/phase-3-peripheral-nervous-system-diseases-4-2021-00c86

Engensis for Painful Diabetic Peripheral Neuropathy · Info ... The meta-analysis of 8 studies involving 656 patients showed that Tongmai Jiangtang capsules (TJC) combined with conventional treatment significantly improved ...

6. trial.medpath.com trial.medpath.com/clinical-trial/c10332d18ca528dc/nct01002235-safety-tolerability-study-vm202-diabetic-neuropathy

Gene Therapy for Painful Diabetic Neuropathy | MedPath The purpose of this open label, dose-escalation, 3-cohort study is to assess the safety and tolerability of injecting Engensis (VM202) in the leg muscle in ...