Extension of Phase 3 Gene Therapy for Painful Diabetic Neuropathy

NCT04055090 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: VMDN-003b
• Study Type: interventional
• Target: 101
• Locations: 1 country
• Sites: 14

Brief Summary

The purpose of this study is to explore the overall safety profile and durability of efficacy of Engensis (VM202) in painful diabetic peripheral neuropathy. All subjects still in follow-up for the VMDN-003 study or who have completed the Day 270 visit within the prior 90 days will be approached to enroll in the long-term safety extension study.

Conditions

Painful Diabetic Neuropathy; Diabetic Neuropathy, Painful

Keywords

diabetic; neuropathy; shooting pain; burning pain; pins and needles pain; foot pain; ViroMed; Helixmith; Engensis; VM202

Outcome Measures

Primary Outcomes (1)

• Long-term Safety for Engensis Versus Placebo

  Long-term (6 months) safety in terms of the incidence of Treatment-emergent Adverse Events and Treatment-emergent Serious Adverse Events for Subjects who received Engensis or Placebo (in the prior VMDN-003 study)

  Baseline through Day 365

Secondary Outcomes (4)

• The Change in the Average 24-hour Pain Score From Baseline (Day 0 of Study VMDN-003) to Day 365 for Engensis Versus Placebo

  Baseline to the Day 365

• Change in the Average 24-hour Pain Score From Day 270 to Day 365 for Engensis Versus Placebo

  Day 270 to Day 365

• Patient's Global Impression of Change at the Day 365 Visit for Engensis Versus Placebo

  At the Day 365 visit

• Subgroup Analysis of the Change in the Average 24-hour Pain Score From Baseline (Day 0 of Study VMDN-003) to Day 365 for Engensis Versus Placebo for Subjects Without Gabapentin and/or Pregabalin Use at Baseline

  Baseline to Day 365

Study Arms (2)

Subjects who received Engensis (VM202)

EXPERIMENTAL

VM202, Engensis

Genetic: Long-Term Follow-Up of Patients who Received Engensis (VM202)

Subjects who received Placebo

PLACEBO COMPARATOR

Placebo, vehicle

Drug: Long-Term Follow-Up of Patients who Received Placebo

Interventions

Long-Term Follow-Up of Patients who Received Engensis (VM202) GENETIC

No study drug is administered in this study. Patients who received Engensis (VM202) in the previous trial (VMDN-003) will remain blinded and were evaluated in this trial for long-term safety and efficacy.

Subjects who received Engensis (VM202)

Long-Term Follow-Up of Patients who Received Placebo DRUG

No study drug is administered in this study. Patients who received Placebo in the previous trial (VMDN-003) will remain blinded and were evaluated in this trial for long-term safety and efficacy.

Subjects who received Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Were randomized and dosed in the VMDN-003 study
• Received all intramuscular injections of study drug on Days 0, 14, 90, and 104 in the VMDN-003 study
• Were in follow-up for the VMDN-003 study or had completed Day 270 within the last 90 days prior to signing consent

You may not qualify if:

• Were using an investigational drug or treatment
• Were unable or unwilling to give informed consent

Sponsors & Collaborators

• Helixmith Co., Ltd.: lead

Study Sites (14)

Arizona Research Center

Phoenix, Arizona, 85023, United States

Location

Clinical Trials, Inc.

Little Rock, Arkansas, 72205, United States

Location

Northern California Research

Sacramento, California, 95821, United States

Location

Center for Clinical Research

San Francisco, California, 94115, United States

Location

Neurological Research Institute

Santa Monica, California, 90404, United States

Location

Diablo Clinical Research, Inc.

Walnut Creek, California, 94598, United States

Location

Innovative Research of West Florida

Clearwater, Florida, 33756, United States

Location

University of Florida McKnight Brain Institute

Gainesville, Florida, 32611, United States

Location

Clinical Research of West Florida

Tampa, Florida, 33603, United States

Location

The Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Raleigh Neurology Associates, P.A.

Raleigh, North Carolina, 27607, United States

Location

Nerve and Muscle Center of Texas

Houston, Texas, 77030, United States

Location

EVMS (Eastern Virginia Medical School)

Norfolk, Virginia, 23510, United States

Location

Rainier Clinical Research Center, Inc.

Renton, Washington, 98057, United States

Location

MeSH Terms

Conditions

Diabetic Neuropathies; Pain

Condition Hierarchy (Ancestors)

Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases; Diabetes Complications; Diabetes Mellitus; Endocrine System Diseases; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Jinsub Lee, PhD.
• Organization: Helixmith Co., Ltd.

jinsub.lee@helixmith.com

+82-10-8256-0439

Study Officials

• John A. Kessler, MD

  Northwestern University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Long term, prospective, non-interventional, safety extension study of phase 3 trial. Double blind, randomized, placebo-controlled, multicenter study

Study Record Dates

• First Submitted: February 22, 2019
• First Posted: August 13, 2019
• Study Start: February 4, 2019
• Primary Completion: July 24, 2019
• Study Completion: July 24, 2019
• Last Updated: October 9, 2025
• Results First Posted: March 8, 2023

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (14)