Extension of Phase 3 Gene Therapy for Painful Diabetic Neuropathy

Helixmith

Status and phase: Completed; Phase 3

Conditions: Painful Diabetic Neuropathy; Diabetic Neuropathy, Painful

Treatments: Drug: Long-Term Follow-Up of Patients who Received Placebo; Genetic: Long-Term Follow-Up of Patients who Received Engensis (VM202)

Study type: Interventional

Funder types: Industry

Identifiers

NCT04055090

VMDN-003b

Details and patient eligibility

About

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.

Full 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.

Enrollment: 101 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

101 participants in 2 patient groups, including a placebo group

Subjects who received Engensis (VM202)

Experimental group

Description:

VM202, Engensis

Treatment:

Genetic: Long-Term Follow-Up of Patients who Received Engensis (VM202)

Subjects who received Placebo

Placebo Comparator group

Description:

Placebo, vehicle

Treatment:

Drug: Long-Term Follow-Up of Patients who Received Placebo