Extension of Phase 3 Gene Therapy for Painful Diabetic Neuropathy

Long-term, Prospective, Non-interventional, Safety Extension of a Phase III, Double-blind, Randomized, Placebo-controlled, Multicenter Study to Assess the Safety & Efficacy of Engensis (VM202) in Subjects With Painful Diabetic Peripheral Neuropathy

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.

Study Overview

• Status: Completed
• Conditions: Diabetic Neuropathy, Painful; Painful Diabetic Neuropathy
• Intervention / Treatment: Genetic: Long-Term Follow-Up of Patients who Received Engensis (VM202); Drug: Long-Term Follow-Up of Patients who Received Placebo

Detailed Description

In the phase III VMDN-003 study, subjects received 2 treatments of either Engensis (VM202) or placebo administered as intramuscular (IM) 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 HGF make long-term follow-up important both for safety and efficacy: in order for Engensis to be a candidate for chronic treatment of PDPN, 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.

• Study Type: Interventional
• Enrollment (Actual): 101
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85023
      • Arizona Research Center
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • Clinical Trials, Inc.
  • California
    • Sacramento, California, United States, 95821
      • Northern California Research
    • San Francisco, California, United States, 94115
      • Center for Clinical Research
    • Santa Monica, California, United States, 90404
      • Neurological Research Institute
    • Walnut Creek, California, United States, 94598
      • Diablo Clinical Research, Inc.
  • Florida
    • Clearwater, Florida, United States, 33756
      • Innovative Research of West Florida
    • Gainesville, Florida, United States, 32611
      • University of Florida McKnight Brain Institute
    • Tampa, Florida, United States, 33603
      • Clinical Research of West Florida
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • The Brigham and Women's Hospital
  • North Carolina
    • Raleigh, North Carolina, United States, 27607
      • Raleigh Neurology Associates, P.A.
  • Texas
    • Houston, Texas, United States, 77030
      • Nerve and Muscle Center of Texas
  • Virginia
    • Norfolk, Virginia, United States, 23510
      • EVMS (Eastern Virginia Medical School)
  • Washington
    • Renton, Washington, United States, 98057
      • Rainier Clinical Research Center, Inc.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Were randomized and dosed in the VMDN-003 study
2. Received all IM 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Subjects who received Engensis (VM202); VM202- Engensis	Genetic: Long-Term Follow-Up of Patients who Received Engensis (VM202); No study drug is administered in this study. Patients who received Engensis (VM202) in a previous trial will be evaluated in this trial for long-term safety and efficacy.
Placebo Comparator: Subjects who received Placebo; Placebo, vehicle	Drug: Long-Term Follow-Up of Patients who Received Placebo; No study drug is administered in this study. Patients who received Placebo in a previous trial will be evaluated in this trial for long-term safety and efficacy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The difference in long-term safety	Defined as occurrence of TEAEs - observed between Participants receiving Engensis versus Participants receiving Placebo in the VMDN-003 study	Baseline through Day 365 follow up

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The change in the average 24-hour pain score from baseline (Day 0 of Study VMDN-003)	The change in the average 24-hour pain score from baseline (Day 0 of Study VMDN-003) to the Day 365 follow-up obtained from the Daily Pain and Sleep Interference Diary	Baseline to the Day 365 follow-up
The change in the average 24-hour pain score from Day 270 to the Day 365 follow-up	The change in the average 24-hour pain score from Day 270 to the Day 365 follow-up from the Daily Pain and Sleep Interference Diary	Day 270 to the Day 365 follow-up
Patient's Global Impression of Change (PGIC) at the Day 365 follow-up	The Patient's Global Impression of Change	At the Day 365 follow-up

Collaborators and Investigators

• Sponsor: Helixmith Co., Ltd.
• Investigators: Principal Investigator: John A. Kessler, MD, Northwestern University

Study record dates

Study Major Dates

• Study Start (Actual): February 4, 2019
• Primary Completion (Actual): July 24, 2019
• Study Completion (Actual): July 24, 2019

Study Registration Dates

• First Submitted: February 22, 2019
• First Submitted That Met QC Criteria: August 12, 2019
• First Posted (Actual): August 13, 2019

Study Record Updates

• Last Update Posted (Actual): September 14, 2022
• Last Update Submitted That Met QC Criteria: September 12, 2022
• Last Verified: September 1, 2022

More Information

Terms related to this study

• Keywords: neuropathy; foot pain; diabetic; shooting pain; burning pain; pins and needles pain; ViroMed; VM202; Engensis; Helixmith
• Additional Relevant MeSH Terms: Nervous System Diseases; Neurologic Manifestations; Endocrine System Diseases; Diabetes Complications; Diabetes Mellitus; Neuromuscular Diseases; Peripheral Nervous System Diseases; Pain; Diabetic Neuropathies
• Other Study ID Numbers: VMDN-003b

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No