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Completed

NCT Number: NCT04055090

Extension of Phase 3 Gene Therapy for Painful Diabetic 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.

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Key information

Conditions

Painful Diabetic Neuropathy Diabetes Complications Diabetes Mellitus Diabetic Neuropathies Diabetic Neuropathy, Painful Endocrine System Diseases Nervous System Diseases Neurologic Manifestations Neuromuscular Diseases Pain Pathological Conditions, Signs and Symptoms Peripheral Nervous System Diseases Signs and Symptoms

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Arizona Research Center, Phoenix, Arizona, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

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

Treatment and study plan

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.

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.

Primary outcomes

  1. Long-term Safety for Engensis Versus Placebo

    Time frame: Baseline through Day 365

    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)

Secondary outcomes

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

    Time frame: Baseline to the Day 365

    The Average 24-hour Pain Score was obtained from the Daily Pain and Sleep Interference Diary. The change in the Average 24-hour Pain Score was determined from baseline (Day 0 of Study VMDN-003) to the Day 365 visit. The Average 24-hour Pain Score is an 11-point numerical scale with scores from 0 (No Pain) to 10 (Worst Possible Pain).

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

    Time frame: Day 270 to Day 365

    The Average 24-hour Pain Score is from the Daily Pain and Sleep Interference Diary. The change in the Average 24-hour Pain Score was determined for Day 270 to Day 365. The Average 24-hour Pain Score is an 11-point numerical scale with scores from 0 (No Pain) to 10 (Worst Possible Pain).

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

    Time frame: At the Day 365 visit

    The Patient's Global Impression of Change was completed by subjects (self-administered) at the Day 365 visit. The subject evaluated how his/her overall status had changed since the start of the study using a 7-point Patient's Global Impression of Change questionnaire scale, where 1 = Very Much Improved, 2 = Much Improved, 3 = Minimally Improved, 4 = No Change, 5 = Minimally Worse, 6 = Much Worse, and 7 = Very Much Worse.

    The Outcome Measure was the Patient's Global Impression of Change Categories of Scores as follows: 1 = Very Much Improved or Much Improved, 0 = Minimally Improved/Worsened or No Change, and -1 = Much Worse or Very Much Worse.

  4. 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

    Time frame: Baseline to Day 365

    The Average 24-hour Pain Score was obtained from the Daily Pain and Sleep Interference Diary and the change in the Average 24-hour Pain Score from baseline (Day 0 of Study VMDN-003) to the Day 365 follow-up was determined. The Average 24-hour Pain Score is an 11-point numerical scale with scores from 0 (No Pain) to 10 (Worst Possible Pain).

Sponsors and collaborators

Lead sponsor

Helixmith Co., Ltd.

Industry

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2019
Study completion
2019
First posted
Aug 13, 2019
Registry last updated
Oct 9, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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