Study to Assess Safety and Efficacy of Engensis in Painful Diabetic Peripheral Neuropathy

NCT04469270 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: VMDN-003-2
• Study Type: interventional
• Target: 162
• Locations: 1 country
• Sites: 16

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of intramuscular administration of Engensis on pain in participants with painful diabetic peripheral neuropathy in the feet and lower legs, as compared to Placebo, as a second Phase 3, well controlled study, sufficient in supporting the efficacy and safety of Engensis.

Conditions

Diabetic Neuropathy, Painful

Outcome Measures

Primary Outcomes (1)

• Efficacy of Engensis Compared to Placebo Painful Diabetic Peripheral Neuropathy in Feet and Lower Legs Comparing Average Daily Pain Score From Day 0 Visit to Day 180 Visit on Brief Pain Inventory for Participants With Diabetic Peripheral Neuropathy

  • The Brief Pain Inventory for Participants with Diabetic Peripheral Neuropathy has a minimum score of 0 and a maximum score of 10, with a higher score representing a worse outcome of more pain. Change in Baseline to Day 180. Summary of the Actual Value of the Change from Baseline to Day 180 in Average Daily Pain Score (Intent-To-Treat Population). Overall Engensis n=79.

  180 days

Secondary Outcomes (5)

• Efficacy of Engensis on Worst Pain in Painful Diabetic Peripheral Neuropathy in Feet and Legs by Comparing Change From Baseline (Day 0) in Worst Pain Score From Brief Pain Inventory for Diabetic Peripheral Neuropathy to Day 180 Compared to Placebo

  180 days

• Efficacy of Engensis Reducing Painful Diabetic Peripheral Neuropathy in Feet and Legs by Determining a ≥ 50% Reduction in the Average Daily Pain Score From Baseline to Day 180 Using the Brief Pain Inventory With Participants Diabetic Peripheral Neuropathy

  180 days

• Safety of Engensis in Painful Diabetic Peripheral Neuropathy in Feet and Legs Comparing Incidence of Adverse and Serious Adverse Events, Incidence of Injection Site Reactions, and Incidence of Clinically Significant Laboratory Values to Placebo

  180 days

• To Evaluate the Possibility of Cellular Responses to Engensis

  104 days

• To Evaluate the Possibility of Humoral Responses to Engensis - Anti-Hepatic Growth Factor

  Days 0, 60, 90, 150 and 180

Study Arms (2)

Engensis

EXPERIMENTAL

16 (ea) 0.25mg (0.5 mL) injections in each of the right and left gastrocnemius muscles on Days 0, 14, 90, and 104.

Biological: Engensis

Placebo

PLACEBO COMPARATOR

16 0.5 mL injections in each of the right and left gastrocnemius muscles on Days 0, 14, 90, and 104.

Other: Placebo

Interventions

Engensis BIOLOGICAL

Intramuscular injections

Engensis

Placebo OTHER

Intramuscular injections

Placebo

Eligibility Criteria

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

You may qualify if:

• Male or female participants age ≥ 18 years at time of completion of the informed consent process
• Type 1 or 2 diabetes mellitus and on current Standards of Medical Care in Diabetes - 2020 optimal guideline-directed medical therapy in participants (including vaccine recommendations if possible), and without unstable diabetes or significant medical problems, such as progressive end-organ disease, within 3 months of or during Screening, in the judgment of the Investigator
• Glycosylated HbA1c of ≤ 10.0% using the first assessment collected during Screening
• Documented diagnosis of bilateral painful diabetic peripheral neuropathy in both lower extremities at least 6 months prior to Screening
• An Average Daily Pain Score ≥ 4 (standard deviation ≥ 0.3 and ≤ 1.5) that was completed during the 7 days prior to randomization (Day 0)
• The physical examination component of the Michigan Neuropathy Screening Instrument score of ≥ 2.5
• If on medication for painful diabetic peripheral neuropathy (other than gabapentin or pregabalin), must have been on a stable dose defined as \< 50% change in total dose over 3 months prior to completion of informed consent
• Male participants and their female partners had to agree to use double-barrier contraception during the study or provide proof of postmenopausal state (minimum 1 year) or surgical sterility
• Male participants were not to donate sperm during the study
• Female participants had to be nonpregnant, nonlactating, and either postmenopausal for at least 1 year, or surgically sterile for at least 3 months, or agreed to use double-barrier contraception from 28 days prior to randomization and/or their last confirmed menstrual period prior to study randomization (whichever is longer) until the end of the study
• Capable and willing to comply with the requirements and restrictions of the protocol and informed consent form
• Able to complete all screening activities within 52 days of signing the informed consent form.

You may not qualify if:

• Other sources of pain that prevented accurate assessment of diabetic peripheral neuropathy pain (e.g., thoracic and/or lumbar root proximal neuropathy, mononeuritis multiplex)
• Peripheral neuropathy caused by a condition other than diabetes: e.g., anatomic (sciatic nerve compression), systemic (monoclonal gammopathy), metabolic (thyroid disease), and toxic (alcohol use) neuropathies
• Had taken gabapentin or pregabalin during 30 days before completion of informed consent process or was going to take at any time during the study
• Progressive or degenerative neurological disorder, such as amyotrophic lateral sclerosis, Alzheimer's disease, Parkinson's disease, vascular dementia, multiple sclerosis, or other neurological disorders determined by the Investigator to preclude participation
• Symptomatic peripheral artery disease or peripheral artery disease requiring revascularization and/or that may interfere with the conduct of the study
• Vasculitis, such as from Buerger's or other diseases
• Systolic blood pressure \>180 mmHg on tolerable doses of standard antihypertensive medications at Screening determined by the Investigator to preclude participation
• Hyperlipidemia or dyslipidemia not being treated with an optimal treatment regimen that follows the Standards of Care for hyperlipidemic/dyslipidemic patients with DM
• Class 3 or 4 heart failure
• Symptomatic bradycardia or untreated high degree atrioventricular block
• Stroke or cerebrovascular accident or myocardial infarction within 3 months before Screening
• Estimated glomerular filtration rate \< 30 mL/min/1.73 m2 using the chronic kidney disease epidemiology collaboration formula based on Cystatin C levels
• Progressive renal dysfunction, defined as a decrease in estimated glomerular filtration rate to chronic kidney disease Stage 1, 2, or 3 in the past 6 months before Screening
• Ophthalmologic conditions pertinent to signs or symptoms of proliferative diabetic retinopathy or other ocular conditions that precluded standard ophthalmologic examination
• Myopathy (e.g., Duchenne or Becker muscular dystrophy, polymyositis)

Sponsors & Collaborators

• Helixmith Co., Ltd.: lead

Study Sites (16)

Arizona Research Center

Phoenix, Arizona, 85053, United States

Location

Clinical Trials - Little Rock

Little Rock, Arkansas, 72205, United States

Location

California Medical Clinic for Headache

Los Angeles, California, 90048, United States

Location

Clinical Trials Research - Sacramento

Sacramento, California, 95821, United States

Location

Innovative Research of West Florida, Inc.

Clearwater, Florida, 33756, United States

Location

Gateway Clinical Trials, LLC

O'Fallon, Illinois, 62269, United States

Location

Foot & Ankle Center of Illinois

Springfield, Illinois, 62704, United States

Location

Clinical Research Professionals

Chesterfield, Missouri, 63005, United States

Location

Richmond Behavioral Associates

Staten Island, New York, 10314, United States

Location

Health Concepts

Rapid City, South Dakota, 57702, United States

Location

Nerve and Muscle Center of Texas

Houston, Texas, 77030, United States

Location

Futuro Clinical Trials, LLC

McAllen, Texas, 78501, United States

Location

ClinPoint Trials LLC

Waxahachie, Texas, 75165, United States

Location

Manassas Clinical Research Center

Manassas, Virginia, 20110, United States

Location

Eastern Virginia Medical School

Norfolk, Virginia, 23510, United States

Location

Dominion Medical Associates

Richmond, Virginia, 23219, United States

Location

MeSH Terms

Conditions

Diabetic Neuropathies

Condition Hierarchy (Ancestors)

Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases; Diabetes Complications; Diabetes Mellitus; Endocrine System Diseases

Results Point of Contact

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

jinsub.lee@helixmith.com

+82-10-8256-0439

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
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 25, 2020
• First Posted: July 14, 2020
• Study Start: November 20, 2020
• Primary Completion: March 24, 2023
• Study Completion: July 31, 2024
• Last Updated: October 9, 2025
• Results First Posted: January 20, 2025

Record last verified: 2025-08

Locations

US (16)