Gene Therapy for Painful Diabetic Neuropathy

NCT01002235 | Completed Phase 1 Results DMC

• 1 other identifier: VMDN-001/G
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of this open label, Phase I/II, dose-escalation, 3-cohort, multicenter, 12-month study, is to assess the safety and tolerability of injecting Engensis (VM202) in the leg muscle in patients with painful diabetic peripheral neuropathy (DPN). The study will also assess the potential of VM202 to reduce the pain associated with diabetic peripheral neuropathy.

Conditions

Painful Diabetic Peripheral Neuropathy

Keywords

Diabetic; Neuropathy; Peripheral; diabetes

Outcome Measures

Primary Outcomes (1)

• Treatment-emergent Adverse Events for Participants Who Received Engensis 4 mg, 8 mg, or 16 mg

  Treatment-emergent adverse events are any adverse events that occurred after the first dose of Engensis (VM202). This is a dose escalation study. Cohorts of increasing dose will be enrolled sequentially. Dose escalation decisions (permission to treat at higher doses) will be made by the Data Safety Monitoring Board based on review of adverse events and on the occurrence of dose limiting toxicities in each cohort. The decision to proceed to the next higher dose cohort will be made according to the scheme described in the protocol.

  Day 0 to Day 365

Secondary Outcomes (1)

• Percent Change From Baseline in Visual Analog Scale Pain Scores

  Days 0, 90, 180, and 365

Study Arms (3)

Cohort 1

EXPERIMENTAL

Two divided doses of VM202 injected into the calf muscle on Day 0 and Day 14 for a total dose of 4 mg.

Biological: VM202

Cohort 2

EXPERIMENTAL

Two divided doses of VM202 injected into the calf muscle on Day 0 and Day 14 for a total dose of 8mg.

Biological: VM202

Cohort 3

EXPERIMENTAL

Two divided doses of VM202 injected into the calf muscle on Day 0 and Day 14 for a total dose of 16mg.

Biological: VM202

Interventions

VM202 BIOLOGICAL

Intramuscular injections in the calf on Day 0 and Day 14.

Cohort 1; Cohort 2; Cohort 3

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%)
• Diagnosis of painful diabetic peripheral neuropathy in both lower extremities
• The physical examination component of the Michigan Neuropathy Screening Instrument Score (MNSI) is ≥ 3 at Screening
• Visual analog scale (VAS) score of ≥ 4 cm at Screening (0 cm = no pain - 10 cm worst imaginable pain)
• Stable treatment of diabetes for at least 3 months with no anticipated changes in medication regimen, and no new symptoms associated with diabetes
• Lower extremity pain for at least 6 months
• If female of childbearing potential, negative 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, Rheumatoid Arthritis)
• Positive HIV or HTLV at Screening
• Positive 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 or known immunosuppression or on chronic treatment with immunosuppressive drugs, chemotherapy or radiation therapy
• Stroke or myocardial infarction within last 6 months;
• Ophthalmologic conditions pertinent to proliferative retinopathy or conditions that preclude standard ophthalmologic examination:
• Cataract surgery within 6 months of trial;
• Vascular lesions of the anterior segment of the eye (infection or ulceration of the cornea, rubeotic glaucoma, etc);
• Vascular lesions of the posterior segment of the eye or proliferative retinopathy, macular edema, s/p photocoagulation for macular edema or proliferative retinopathy; sickle cell retinopathy, ischemic retinopathy due to retinal venous stasis or carotid artery disease;
• Choroidal angiogenesis; and

Sponsors & Collaborators

• Helixmith Co., Ltd.: lead

Study Sites (2)

Diablo Clinical Research Hospital

Walnut Creek, California, 94598, United States

Location

Northwestern Memorial Hospital

Chicago, Illinois, 60611, United States

Location

Related Publications (1)

• Ajroud-Driss S, Christiansen M, Allen JA, Kessler JA. Phase 1/2 open-label dose-escalation study of plasmid DNA expressing two isoforms of hepatocyte growth factor in patients with painful diabetic peripheral neuropathy. Mol Ther. 2013 Jun;21(6):1279-86. doi: 10.1038/mt.2013.69. Epub 2013 Apr 23.

  PMID: 23609019 BACKGROUND

MeSH Terms

Conditions

Diabetes Mellitus

Condition Hierarchy (Ancestors)

Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases

Results Point of Contact

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

jinsub.lee@helixmith.com

+82-10-8256-0439

Study Officials

• John Kessler, M.D.

  Northwestern Memorial Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Dose Ranging

Study Record Dates

• First Submitted: October 24, 2009
• First Posted: October 27, 2009
• Study Start: February 1, 2010
• Primary Completion: April 1, 2011
• Study Completion: April 1, 2012
• Last Updated: October 6, 2025
• Results First Posted: June 15, 2025

Record last verified: 2025-08

Locations

US (2)