Extension of Phase 3 Gene Therapy for Painful Diabetic Neuropathy

Painful Diabetic Neuropathy Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04055090
• Other study ID #: VMDN-003b
• Status: Completed
• Phase: Phase 3
• Start date: February 4, 2019
• Est. completion date: July 24, 2019

Study information

• Verified date: March 2023
• Source: Helixmith Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

In the phase III VMDN-003 study, subjects received 2 treatments of either Engensis (VM202) or placebo administered as intramuscular (IM) 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 HGF make long-term follow-up important both for safety and efficacy: in order for Engensis to be a candidate for chronic treatment of PDPN, 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 101
• Est. completion date: July 24, 2019
• Est. primary completion date: July 24, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

Inclusion Criteria:

1. Were randomized and dosed in the VMDN-003 study

2. Received all IM 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

Related Conditions & MeSH terms

• Diabetic Neuropathies
• Diabetic Neuropathy, Painful
• Pain
• Painful Diabetic Neuropathy
• Peripheral Nervous System Diseases

Intervention

Genetic:
• Long-Term Follow-Up of Patients who Received Engensis (VM202)
No study drug is administered in this study. Patients who received Engensis (VM202) in the previous trial (VMDN-003) will remain blinded and were evaluated in this trial for long-term safety and efficacy.

Drug:
• Long-Term Follow-Up of Patients who Received Placebo
No study drug is administered in this study. Patients who received Placebo in the previous trial (VMDN-003) will remain blinded and were evaluated in this trial for long-term safety and efficacy.

Locations

Country	Name	City	State
United States	The Brigham and Women's Hospital	Boston	Massachusetts
United States	Innovative Research of West Florida	Clearwater	Florida
United States	University of Florida McKnight Brain Institute	Gainesville	Florida
United States	Nerve and Muscle Center of Texas	Houston	Texas
United States	Clinical Trials, Inc.	Little Rock	Arkansas
United States	EVMS (Eastern Virginia Medical School)	Norfolk	Virginia
United States	Arizona Research Center	Phoenix	Arizona
United States	Raleigh Neurology Associates, P.A.	Raleigh	North Carolina
United States	Rainier Clinical Research Center, Inc.	Renton	Washington
United States	Northern California Research	Sacramento	California
United States	Center for Clinical Research	San Francisco	California
United States	Neurological Research Institute	Santa Monica	California
United States	Clinical Research of West Florida	Tampa	Florida
United States	Diablo Clinical Research, Inc.	Walnut Creek	California

Sponsors (1)

Lead Sponsor	Collaborator
Helixmith Co., Ltd.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Long-term Safety for Engensis Versus Placebo	Long-term (6 months) safety in terms of the incidence of Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) for Subjects who received Engensis or Placebo (in the prior VMDN-003 study)	Baseline through Day 365
Secondary	The Change in the Average 24-hour Pain Score From Baseline (Day 0 of Study VMDN-003) to Day 365 for Engensis Versus Placebo	The Average 24-hour Pain Score was obtained from the Daily Pain and Sleep Interference Diary. The change in the Average 24-hour Pain Score was determined from baseline (Day 0 of Study VMDN-003) to the Day 365 visit. The Average 24-hour Pain Score is an 11-point numerical scale with scores from 0 (No Pain) to 10 (Worst Possible Pain).	Baseline to the Day 365
Secondary	Change in the Average 24-hour Pain Score From Day 270 to Day 365 for Engensis Versus Placebo	The Average 24-hour Pain Score is from the Daily Pain and Sleep Interference Diary. The change in the Average 24-hour Pain Score was determined for Day 270 to Day 365. The Average 24-hour Pain Score is an 11-point numerical scale with scores from 0 (No Pain) to 10 (Worst Possible Pain).	Day 270 to Day 365
Secondary	Patient's Global Impression of Change (PGIC) at the Day 365 Visit for Engensis Versus Placebo	The Patient's Global Impression of Change (PGIC) was completed by subjects (self-administered) at the Day 365 visit. The subject evaluated how his/her overall status had changed since the start of the study using a 7-point PGIC questionnaire scale, where 1 = Very Much Improved, 2 = Much Improved, 3 = Minimally Improved, 4 = No Change, 5 = Minimally Worse, 6 = Much Worse, and 7 = Very Much Worse.; The Outcome Measure was the PGIC Categories of Scores as follows: 1 = Very Much Improved or Much Improved, 0 = Minimally Improved/Worsened or No Change, and -1 = Much Worse or Very Much Worse.	At the Day 365 visit
Secondary	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	The Average 24-hour Pain Score was obtained from the Daily Pain and Sleep Interference Diary and the change in the Average 24-hour Pain Score from baseline (Day 0 of Study VMDN-003) to the Day 365 follow-up was determined. The Average 24-hour Pain Score is an 11-point numerical scale with scores from 0 (No Pain) to 10 (Worst Possible Pain).	Baseline to Day 365