An Adaptive, Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Safety and Efficacy of Engensis in Participants With Painful Diabetic Peripheral Neuropathy
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Helixmith Co., Ltd.
- Enrollment
- 162
- Locations
- 16
- Primary Endpoint
- Efficacy of Engensis Compared to Placebo Painful Diabetic Peripheral Neuropathy in Feet and Lower Legs Comparing Average Daily Pain Score From Day 0 Visit to Day 180 Visit on Brief Pain Inventory for Participants With Diabetic Peripheral Neuropathy
Study Overview
Brief Summary
The purpose of this study is to evaluate the efficacy and safety of intramuscular administration of Engensis on pain in participants with painful diabetic peripheral neuropathy in the feet and lower legs, as compared to Placebo, as a second Phase 3, well controlled study, sufficient in supporting the efficacy and safety of Engensis.
Detailed Description
Overall Design VMDN-003-2 is an adaptive Phase 3, double-blind, randomized, placebo-controlled, multicenter study designed to assess the efficacy and safety of Engensis (containing the active pharmaceutical ingredient VM202) in Participants with painful Diabetic Peripheral Neuropathy. Following completion of the informed consent process, Screening activities (during 45 days [from Day -52 to Day -7] prior to Day 0) will determine which Participants meet all-but-one eligibility criteria, which are assessed by an adjudication procedure, followed by completion of a 7-day eDiary prior to Day 0. Eligible participants will be enrolled and randomly assigned in a double-blind fashion and in a 1:1 ratio on Day 0 to either Engensis or Placebo. During Screening, medical history and familial cancer history, demographics, vital signs, height, body mass index, waist size, physical examination, retinal fundoscopy (by an ophthalmologist), 12-lead electrocardiogram, ultrasound of the right and left gastrocnemius muscles (to guide Study Injections), laboratory assessments, estimated glomerular filtration rate, Hemoglobin A1c levels, viral screening, a record of all concomitant medications and procedures, urine drug analysis, and urine pregnancy test for females of childbearing potential will be conducted.
In addition, the following procedures will be conducted during Screening: Hospital Anxiety and Depression Scale, Accurate Pain Reporting and Placebo Response Reduction, Michigan Neuropathy Screening Instrument, and cancer screening tests.
During 7 days before Day 0 and randomization, Participants must complete the full Brief Pain Inventory for Diabetic Peripheral Neuropathy on an eDiary for determining the Average Daily Pain Scores for at least 5 out of the 7 days. Adverse event assessments will start upon completion of the consent process at the start of Screening.
At any time prior to dosing on Day 0, Bedside Sensory Testing should be administered.
Following randomization, and prior to the first intramuscular injections of Engensis or Placebo on Day 0, the partial Brief Pain Inventory for Diabetic Peripheral Neuropathy, , and quality of life instruments will be completed. Blood will be collected for testing of selected cytokines, anti-Hepatic growth factor antibodies, and laboratory assessments.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Engensis
16 (ea) 0.25mg (0.5 mL) injections in each of the right and left gastrocnemius muscles on Days 0, 14, 90, and 104.
Intervention: Engensis (Biological)
Placebo
16 0.5 mL injections in each of the right and left gastrocnemius muscles on Days 0, 14, 90, and 104.
Intervention: Placebo (Other)
Outcomes
Primary Outcomes
Efficacy of Engensis Compared to Placebo Painful Diabetic Peripheral Neuropathy in Feet and Lower Legs Comparing Average Daily Pain Score From Day 0 Visit to Day 180 Visit on Brief Pain Inventory for Participants With Diabetic Peripheral Neuropathy
Time Frame: 180 days
• The Brief Pain Inventory for Participants with Diabetic Peripheral Neuropathy has a minimum score of 0 and a maximum score of 10, with a higher score representing a worse outcome of more pain. Change in Baseline to Day 180. Summary of the Actual Value of the Change from Baseline to Day 180 in Average Daily Pain Score (Intent-To-Treat Population). Overall Engensis n=79.
Secondary Outcomes
- Efficacy of Engensis on Worst Pain in Painful Diabetic Peripheral Neuropathy in Feet and Legs by Comparing Change From Baseline (Day 0) in Worst Pain Score From Brief Pain Inventory for Diabetic Peripheral Neuropathy to Day 180 Compared to Placebo(180 days)
- Efficacy of Engensis Reducing Painful Diabetic Peripheral Neuropathy in Feet and Legs by Determining a ≥ 50% Reduction in the Average Daily Pain Score From Baseline to Day 180 Using the Brief Pain Inventory With Participants Diabetic Peripheral Neuropathy(180 days)
- Safety of Engensis in Painful Diabetic Peripheral Neuropathy in Feet and Legs Comparing Incidence of Adverse and Serious Adverse Events, Incidence of Injection Site Reactions, and Incidence of Clinically Significant Laboratory Values to Placebo(180 days)
- To Evaluate the Possibility of Cellular Responses to Engensis(104 days)
- To Evaluate the Possibility of Humoral Responses to Engensis - Anti-Hepatic Growth Factor(Days 0, 60, 90, 150 and 180)