Painful Diabetic NeuropathyPhase 3NCT04055090
Extension of Phase 3 Gene Therapy for Painful Diabetic Neuropathy
A Phase 3 study testing Long-Term Follow-Up of Patients who Received Engensis (VM202) in people with Painful Diabetic Neuropathy and Diabetic Neuropathy, Painful, run by Helixmith Co., Ltd. at 14 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Painful Diabetic Neuropathy, Diabetic Neuropathy, Painful
- What is being tested?
- Long-Term Follow-Up of Patients who Received Engensis (VM202)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 101 participants
- Where
- 14 sitesUnited States
- Study dates
- 4 Feb 2019to 24 Jul 2019
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Were using an investigational drug or treatment
- Were unable or unwilling to give informed consent
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
The Brigham and Women's Hospital
Boston, Massachusetts, 02115
Innovative Research of West Florida
Clearwater, Florida, 33756
University of Florida McKnight Brain Institute
Gainesville, Florida, 32611
Nerve and Muscle Center of Texas
Houston, Texas, 77030
Clinical Trials, Inc.
Little Rock, Arkansas, 72205
EVMS (Eastern Virginia Medical School)
Norfolk, Virginia, 23510
Arizona Research Center
Phoenix, Arizona, 85023
Raleigh Neurology Associates, P.A.
Raleigh, North Carolina, 27607
Rainier Clinical Research Center, Inc.
Renton, Washington, 98057
Northern California Research
Sacramento, California, 95821
Center for Clinical Research
San Francisco, California, 94115
Neurological Research Institute
Santa Monica, California, 90404
Clinical Research of West Florida
Tampa, Florida, 33603
Diablo Clinical Research, Inc.
Walnut Creek, California, 94598
About this study
Official 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
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.
Read the detailed description
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.
- Being tested
- Long-Term Follow-Up of Patients who Received Engensis (VM202), Long-Term Follow-Up of Patients who Received Placebo
- Keywords
- diabetic, neuropathy, shooting pain, burning pain, pins and needles pain, foot pain, ViroMed, Helixmith, Engensis, VM202
- Medical subject headings
- Diabetic Neuropathies, Pain
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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 PlaceboDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Subjects who received Engensis (VM202) | Experimental | VM202, Engensis Receives: Genetic: Long-Term Follow-Up of Patients who Received Engensis (VM202) |
| Subjects who received Placebo | Placebo comparator | Placebo, vehicle Receives: Drug: Long-Term Follow-Up of Patients who Received Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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
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).
Measured: Baseline to the Day 365
Change in the Average 24-hour Pain Score From Day 270 to Day 365 for Engensis Versus Placebo
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).
Measured: Day 270 to Day 365
Patient's Global Impression of Change at the Day 365 Visit for Engensis Versus Placebo
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.
Measured: 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
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).
Measured: Baseline to Day 365
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2019
- Study start4 Feb 2019
- Primary completion24 Jul 2019
- Study completion24 Jul 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Painful Diabetic NeuropathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Long term, prospective, non-interventional, safety extension study of phase 3 trial. Double blind, randomized, placebo-controlled, multicenter study
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 101 participants (actual)
Sponsor and investigators
- Lead sponsor
- Helixmith Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- John A. Kessler, MD, Principal investigator, Northwestern University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04055090
- Sponsor's protocol ID
- VMDN-003b
- Status last verified
- Aug 2025
- First submitted
- 22 Feb 2019
- Last updated
- 9 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT04055090
- Acronym / sponsor ID
- VMDN-003b
- Keywords
- diabetic, neuropathy, shooting pain, burning pain, pins and needles pain, foot pain, ViroMed, Helixmith
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 101 participants (actual)
- Lead sponsor
- Helixmith Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 4 Feb 2019 (actual)
- Primary completion
- 24 Jul 2019 (actual)
- Study completion
- 24 Jul 2019 (actual)
- First posted
- 13 Aug 2019 (actual)
- Results
- Posted 8 Mar 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Long term, prospective, non-interventional, safety extension study of phase 3 trial. Double blind, randomized, placebo-controlled, multicenter study
| Arm | Type | Receives |
|---|---|---|
| Subjects who received Engensis (VM202) | Experimental | Genetic: Long-Term Follow-Up of Patients who Received Engensis (VM202)VM202, Engensis |
| Subjects who received Placebo | Placebo comparator | Drug: Long-Term Follow-Up of Patients who Received PlaceboPlacebo, vehicle |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (2 listed)
- Were using an investigational drug or treatment
- Were unable or unwilling to give informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Genetic | Subjects who received Engensis (VM202) |
| Long-Term Follow-Up of Patients who Received PlaceboNo 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. | Drug | Subjects who received Placebo |
Primary outcome
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)
4 secondary outcomes
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
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
Patient's Global Impression of Change at the Day 365 Visit for Engensis Versus Placebo
Time frame: 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
Time frame: Baseline to Day 365
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John A. Kessler, MD principal investigator
Northwestern University
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Long-Term Follow-Up of Patients who Received Engensis (VM202) in people with Painful Diabetic Neuropathy and Diabetic Neuropathy, Painful, run by Helixmith Co., Ltd. at 14 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 8 Mar 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Long-term Safety for Engensis Versus Placebo (time frame: Baseline through Day 365). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2025.