Safety and Efficacy Study for the Treatment of Painful Diabetic Neuropathy

NCT01475786 | Completed Phase 2 Results DMC

• 1 other identifier: VMDN-002
• Study Type: interventional
• Target: 104
• Locations: 2 countries
• Sites: 17

Brief Summary

The purpose of this study is to determine if Engensis (VM202) is safe and effective in treating painful diabetic neuropathy.

Conditions

Painful Diabetic Neuropathies

Keywords

nerve pain; diabetic nerve pain; throbbing; burning; painful feet; tingling; numbness; Engensis (VM202)

Outcome Measures

Primary Outcomes (1)

• The Primary Study Endpoint Was the Change in Average 24-hour Pain Score From Baseline to the 6-month Follow-up.

  The difference in the mean change of the 24 hour pain score was compared between the treatment groups and the placebo arm to determine treatment effect. The average pain scores were obtained from the Daily Pain and Sleep Interference Diary (recorded daily by subjects for 7 days during Screening prior to the first round of injections and again, before the 6 month follow-up). Subjects rated their 24-hor daily pain intensity according to the 11-point numerical rating scale from 0 (no pain) to 10 (worst possible pain).

  seven (7) days before 9 Month visit

Study Arms (3)

Low Dose 16mg Engensis (VM202) and Placebo

ACTIVE COMPARATOR

intramuscular injections in each calf for a total of 16mg Engensis (VM202): Day 0 - 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of normal saline 0.5mL / calf Day 14 - 32 injections / calf and 16 injections of normal saline 0.5mL / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf)

Biological: Low Dose: 16 mg Engensis (VM202); Other: Control- Placebo (normal saline)

High Dose 32mg Engensis (VM202)

ACTIVE COMPARATOR

Day 0 - 32 injections of 0.5mL of VM202 / calf (8 mg of VM202 / calf) Day 14 - 32 injections of 0.5mL of VM202 / calf (8 mg of VM202 / calf) For a total of 32mg VM202

Biological: High Dose: 32 mg Engensis (VM202)

Control - Placebo (normal saline)

PLACEBO COMPARATOR

32 injections / calf of 0.5 mL normal saline at Day 0 and Day 14

Other: Control- Placebo (normal saline)

Interventions

Low Dose: 16 mg Engensis (VM202) BIOLOGICAL

Subjects in the Low Dose Group (8mg VM202 / leg) will receive the following intramuscular injections in each calf: Day 0 - 32 injections / calf: • 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) Day 14 - 32 injections / calf: • 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf)

Also known as: Gene Therapy

Low Dose 16mg Engensis (VM202) and Placebo

High Dose: 32 mg Engensis (VM202) BIOLOGICAL

Subjects in the High Dose Group (16 mg VM202 / leg) will receive the following intramuscular injections in each calf: Day 0 • 32 injections of 0.5mL of VM202 / calf (8 mg of VM202 / calf) Day 14 • 32 injections of 0.5mL of VM202 / calf (8 mg of VM202 / calf)

Also known as: Gene Therapy

High Dose 32mg Engensis (VM202)

Control- Placebo (normal saline) OTHER

subjects will receive thirty-two (32) 0.5 mL injections of normal saline on Day 0 and Day 14.

Also known as: Normal saline

Control - Placebo (normal saline); Low Dose 16mg Engensis (VM202) and Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 years to ≤ 75 years
• Documented history of Type I or II diabetes with current treatment control (glycosylated hemoglobin A1c of ≤ 10.0% at Screening) and currently on oral medication and/or insulin
• Diagnosis of painful diabetic peripheral neuropathy in both lower extremities
• Lower extremity pain for at least 6 months
• Visual analog scale (VAS) score of ≥ 40 mm at Initial Screening (0 mm = no pain - 100 mm very severe pain)
• Symptoms from the Brief Pain Neuropathy Screening (BPNS) is ≤ 5 point difference between legs at Initial Screening
• The average daily pain intensity score of the Daily Pain and Sleep Interference Diary completed after medication wash-out is ≥ 4 with a standard deviation ≤ 2
• The physical examination component of the Michigan Neuropathy Screening Instrument Score (MNSI) is ≥ 3 at Screening
• Stable treatment of diabetes for at least 3 months with no anticipated changes in medication regimen, and no new symptoms associated with diabetes
• If female of childbearing potential, negative urine pregnancy test at screening and using acceptable method of birth control during the study

You may not qualify if:

• Peripheral neuropathy caused by condition other than diabetes
• Other pain more severe than neuropathic pain
• Progressive or degenerative neurological disorder
• Myopathy
• Inflammatory disorder of the blood vessels (inflammatory angiopathy, such as Buerger's disease)
• Active infection
• Chronic inflammatory disease (e.g., Crohn's disease, rheumatoid arthritis)
• Positive HIV or HTLV at Screening
• Active Hepatitis B or C as determined by Hepatitis B core antibody (HBcAB), antibody to Hepatitis B antigen (IgG and IgM; HbsAb), Hepatitis B surface antigen (HBsAg) and Hepatitis C antibodies (Anti-HCV) at Screening
• Subjects with known immunosuppression or currently receiving immunosuppressive drugs, chemotherapy or radiation therapy
• Stroke or myocardial infarction within last 3 months
• Ophthalmologic conditions pertinent to proliferative retinopathy or conditions that preclude standard ophthalmologic examination
• Uncontrolled hypertension as defined as sustained systolic blood pressure (SBP) \> 200 mmHg or diastolic BP (DBP) \> 110 mmHg at Screening
• Patients with a recent history (\< 5 years) of or new screening finding of malignant neoplasm except basal cell carcinoma or squamous cell carcinoma of the skin (if excised and no evidence of recurrence); patients with family history of colon cancer in any first degree relative are excluded unless they have undergone a colonoscopy in the last 12 months with negative findings
• Subjects requiring \> 81 mg daily of acetylsalicylic acid; If ≥ 81 mg are taken at screening, subjects may be enrolled if willing/able to switch to another medication

Sponsors & Collaborators

• Helixmith Co., Ltd.: lead

Study Sites (17)

Diablo Clinical Research

Walnut Creek, California, 94598, United States

Location

Compass Research

Orlando, Florida, 32806, United States

Location

Palm Beach Neurological Center

Palm Beach Gardens, Florida, 33418, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Beth Israel Deaconess

Boston, Massachusetts, 02115, United States

Location

The Neurosciences Institute Albany Medical College

Albany, New York, 12208, United States

Location

Mount Sinai Medical Center

New York, New York, 10029, United States

Location

University of Oklahoma Harold Hamm Diabetes Center

Oklahoma City, Oklahoma, 73104, United States

Location

Houston Neurocare

Houston, Texas, 77030, United States

Location

The Methodist Hospital

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

East Virginia Medical School Strelitz Diabetes Center

Norfolk, Virginia, 23510, United States

Location

Rainier Clinical Research

Seattle, Washington, 98057, United States

Location

Seoul National University Bundang Hospital

Seongnam, Bundang-gu, 463-707, South Korea

Location

Yonsei University College of Medicine Severance Hospital

Seoul, Seodaemun-gu, 120-752, South Korea

Location

Sejong General Hospital

Bucheon-si, Sosa-gu, 422-711, South Korea

Location

The Catholic University of Korea Youido St. Mary's Hospital

Seoul, Yeongdeungpo-gu, 150-713, South Korea

Location

Related Publications (1)

• Kessler JA, Smith AG, Cha BS, Choi SH, Wymer J, Shaibani A, Ajroud-Driss S, Vinik A; VM202 DPN-II Study Group. Double-blind, placebo-controlled study of HGF gene therapy in diabetic neuropathy. Ann Clin Transl Neurol. 2015 May;2(5):465-78. doi: 10.1002/acn3.186. Epub 2015 Mar 5.

  PMID: 26000320 RESULT

MeSH Terms

Conditions

Diabetic Neuropathies; Neuralgia; Paresthesia; Hypesthesia

Interventions

Genetic Therapy; Saline Solution

Condition Hierarchy (Ancestors)

Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases; Diabetes Complications; Diabetes Mellitus; Endocrine System Diseases; Pain; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Somatosensory Disorders; Sensation Disorders

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics; Genetic Engineering; Genetic Techniques; Investigative Techniques; Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Results Point of Contact

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

jinsub.lee@helixmith.com

+82-10-8256-0439

Study Officials

• Jack Kessler, MD

  Northwestern University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 15, 2011
• First Posted: November 21, 2011
• Study Start: August 1, 2012
• Primary Completion: March 12, 2014
• Study Completion: June 1, 2014
• Last Updated: October 6, 2025
• Results First Posted: July 3, 2023

Record last verified: 2025-08

Locations

US (13); KR (4)