Extension of Phase 3 Gene Therapy for Painful Diabetic Neuropathy

NCT ID: NCT04055090

Last Updated: 2025-10-09

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

101 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-02-04

Study Completion Date

2019-07-24

Brief Summary

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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.

Detailed Description

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In the phase III VMDN-003 study, subjects received 2 treatments of either Engensis (VM202) or placebo administered as intramuscular injections into bilateral calves on Days 0 and 14, and Days 90 and 104. Primary efficacy was evaluated 90 days following the first injection. The growth potential for Hepatocyte Growth Factor make long-term follow-up important both for safety and efficacy: in order for Engensis to be a candidate for chronic treatment of Painful Diabetic Peripheral Neuropathy, it must be demonstrated not to induce unexpected adverse events with repeated dosing; and the potential for reversal or stabilization of diabetic neuropathy using only one or two treatments of Engensis may make it especially attractive compared to current treatments which must be taken daily for the duration of the disease. A safety extension to the VMDN-003 study is therefore warranted.

Conditions

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Painful Diabetic Neuropathy Diabetic Neuropathy, Painful

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Long term, prospective, non-interventional, safety extension study of phase 3 trial. Double blind, randomized, placebo-controlled, multicenter study
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Double-blind

Study Groups

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Subjects who received Engensis (VM202)

VM202, Engensis

Group Type EXPERIMENTAL

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

Intervention Type 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 Placebo

Placebo, vehicle

Group Type PLACEBO_COMPARATOR

Long-Term Follow-Up of Patients who Received Placebo

Intervention Type 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.

Interventions

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

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.

Intervention Type GENETIC

Long-Term Follow-Up of Patients who Received Placebo

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.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

1. Were using an investigational drug or treatment
2. Were unable or unwilling to give informed consent
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Helixmith Co., Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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John A. Kessler, MD

Role: PRINCIPAL_INVESTIGATOR

Northwestern University

Locations

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Arizona Research Center

Phoenix, Arizona, United States

Site Status

Clinical Trials, Inc.

Little Rock, Arkansas, United States

Site Status

Northern California Research

Sacramento, California, United States

Site Status

Center for Clinical Research

San Francisco, California, United States

Site Status

Neurological Research Institute

Santa Monica, California, United States

Site Status

Diablo Clinical Research, Inc.

Walnut Creek, California, United States

Site Status

Innovative Research of West Florida

Clearwater, Florida, United States

Site Status

University of Florida McKnight Brain Institute

Gainesville, Florida, United States

Site Status

Clinical Research of West Florida

Tampa, Florida, United States

Site Status

The Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status

Raleigh Neurology Associates, P.A.

Raleigh, North Carolina, United States

Site Status

Nerve and Muscle Center of Texas

Houston, Texas, United States

Site Status

EVMS (Eastern Virginia Medical School)

Norfolk, Virginia, United States

Site Status

Rainier Clinical Research Center, Inc.

Renton, Washington, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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VMDN-003b

Identifier Type: -

Identifier Source: org_study_id

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