Clinical trial NCT04055090: Extension of Phase 3 Gene Therapy for Painful Diabetic Neuropathy
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Painful Diabetic Neuropathy
Diabetic Neuropathy, Painful
Keywords: diabetic
neuropathy
shooting pain
burning pain
pins and needles pain
foot pain
ViroMed
Helixmith
Engensis
VM202
Lead sponsor: Helixmith Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2019-08-13
Enrollment: 101
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Long term, prospective, non-interventional, safety extension study of phase 3 trial. Double blind, randomized, placebo-controlled, multicenter study
Primary purpose: TREATMENT

Brief 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 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.

Eligibility criteria: Inclusion Criteria:

1. Were randomized and dosed in the VMDN-003 study
2. Received all intramuscular injections of study drug on Days 0, 14, 90, and 104 in the VMDN-003 study
3. Were in follow-up for the VMDN-003 study or had completed Day 270 within the last 90 days prior to signing consent

Exclusion Criteria:

1. Were using an investigational drug or treatment
2. Were unable or unwilling to give informed consent

Primary outcomes:
measure: Long-term Safety for Engensis Versus Placebo
timeFrame: Baseline through Day 365
Secondary outcomes:
measure: The Change in the Average 24-hour Pain Score From Baseline (Day 0 of Study VMDN-003) to Day 365 for Engensis Versus Placebo
timeFrame: Baseline to the Day 365
measure: Change in the Average 24-hour Pain Score From Day 270 to Day 365 for Engensis Versus Placebo
timeFrame: Day 270 to Day 365
measure: Patient's Global Impression of Change at the Day 365 Visit for Engensis Versus Placebo
timeFrame: At the Day 365 visit
measure: 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
timeFrame: Baseline to Day 365
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Arizona Research Center
state: Arizona
city: Little Rock
country: United States
facility: Clinical Trials, Inc.
state: Arkansas
city: Sacramento
country: United States
facility: Northern California Research
state: California
city: San Francisco
country: United States
facility: Center for Clinical Research
state: California
city: Santa Monica
country: United States
facility: Neurological Research Institute
state: California
city: Walnut Creek
country: United States
facility: Diablo Clinical Research, Inc.
state: California
city: Clearwater
country: United States
facility: Innovative Research of West Florida
state: Florida
city: Gainesville
country: United States
facility: University of Florida McKnight Brain Institute
state: Florida
city: Tampa
country: United States
facility: Clinical Research of West Florida
state: Florida
city: Boston
country: United States
facility: The Brigham and Women's Hospital
state: Massachusetts
city: Raleigh
country: United States
facility: Raleigh Neurology Associates, P.A.
state: North Carolina
city: Houston
country: United States
facility: Nerve and Muscle Center of Texas
state: Texas
city: Norfolk
country: United States
facility: EVMS (Eastern Virginia Medical School)
state: Virginia
city: Renton
country: United States
facility: Rainier Clinical Research Center, Inc.
state: Washington
Study officials:
affiliation: Northwestern University
name: John A. Kessler, MD
role: PRINCIPAL_INVESTIGATOR
References: