Phase 3 Gene Therapy for Painful Diabetic Neuropathy

Summary

The purpose of this study is to determine the safety and efficacy of bilateral intramuscular injections of VM202 versus placebo in the treatment of painful diabetic peripheral neuropathy. A total of 507 of 477 planned participants were randomized in a 2:1 ratio to one of two treatment groups. Note that 500 participants received Investigational product treatment, whereas 7 participants did not receive Investigational product treatment. Treatments - Engensis (VM202) - 336 Engensis of 318 planned participants Control - Placebo (VM202 vehicle) - 164 Placebo of 159 planned participants Randomization were stratified by current use of gabapentin and/or pregabalin.

Timeline

Start

2016-04

Primary completion

2019-04

Completion

2019-04

Outcome

Missed primary endpoint

paper VM202 failed to meet its efficacy end points in DPN 3-1. PMID 33465273 ↗

Publications

• Results Kessler JA, Shaibani A, Sang CN, Christiansen M, Kudrow D, Vinik A, Shin N; VM202 study group. Gene therapy for diabetic peripheral neuropathy: A randomized, placebo-controlled phase III study of VM202, a plasmid DNA encoding human hepatocyte growth factor. Clin Transl Sci. 2021 May;14(3):1176-1184. doi: 10.1111/cts.12977. Epub 2021 Feb 2.

Drugs

Indications