The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)

NCT05361031 | Completed Phase 1 Results

• 1 other identifier: VMCMT-001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

To assess the safety and tolerability of the investigational product Engensis (VM202) injected in the weakened lower limb muscles of CMT1A patients

Conditions

CMT

Outcome Measures

Primary Outcomes (1)

• Safety of Intramuscular (IM) Injections of Engensis in Participants

  Frequency and percentage of treatment-emergent adverse events (TEAEs) and serious adverse events after injections, injection site reactions, vital sign, and clinically significant laboratory values for Engensis.

  Day 0 visit to the Day 270 visit

Secondary Outcomes (7)

• Evaluation of Severity of Disease Changes Following Engensis Injections - Charcot Marie Tooth Neuropathy Score Version 2

  Day 0 and Day 270

• Evaluation of Patient's Neurological Disability - Functional Disability Score

  Day 0, 90, 180 and Day 270

• Evaluation of the Activity Level of Patients With Peripheral Neuropathy Using the Overall Neuropathy Limitation Score Leg Scale

  Day 0, 90, 180 and Day 270

• Evaluation of Patient's Walking Ability - Change From Baseline in the 10-Meter Walk Test

  Day 0, 90, 180 and Day 270

• Changes in Fatty Infiltration Level of Lower Limb Muscles

  Day 0 and Day 270

Study Arms (1)

Engensis (VM202)

EXPERIMENTAL

56 (ea) 0.25mg (0.5 mL) injections in each of the left and right lower limbs on Days 0, 14, 90, and 104.

Biological: Engensis (VM202)

Interventions

Engensis (VM202) BIOLOGICAL

Intramuscular injections

Engensis (VM202)

Eligibility Criteria

• Age: 19 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or females ≥ 19 years of age and ≤ 65 years of age
• Patients with confirmed diagnosis of CMT1A by genetic testing
• Patients with mild to moderate severity assessed by Charcot Marie Tooth Neuropathy Score version 2 (CMTNS v2) with a score \> 2 and ≤ 20
• Individuals with lower limb muscle weakness with minimum dorsiflexion or more
• Individuals who voluntarily consented to participate in this study and signed the IRB approved informed consent form after listening to a description on the characteristics of this clinical study prior to all screening tests
• Individuals who can comply with the requirements in the clinical study
• In case of females of child bearing potential, those who test negative in a urine or serum pregnancy test at screening
• Individuals who practice medically approved contraceptive methods throughout the clinical study

You may not qualify if:

• Patients with significant respiratory, circulatory, renal, gastrointestinal, hepatic, endocrine, hematologic, psychiatric disorders or other severe diseases, or alcohol or drug addiction who may develop safety issues or cause confusion in the interpretation of the clinical study results as determined by the principal investigator
• Patients with other neuromuscular diseases or neuropathy-inducing factors: Patients with chronic alcohol addiction, undergoing anticancer chemotherapy, or taking neurotoxic drugs
• Patients diagnosed with diabetes
• Patients diagnosed with inflammatory bowel disease
• Patients with a history of stroke or cerebral ischemic attack within 12 months prior to the screening date
• Patients with a history of coronary artery disease, such as myocardial infarction and unstable angina pectoris, within 12 months prior to the screening date
• Morbidly obese patients with body mass index (BMI) ≥ 37
• Patients who underwent orthopedic surgery (corrective surgery for bone and ligament, artificial joint implantation, osteosynthesis, osteotomy, arthroscopic surgery) in the lower limbs within 6 months prior to the screening date
• Patients who may be affected by the muscle strength measurement test due to ankle contracture or surgery
• Patients with uncontrolled hypertension (if systolic blood pressure is ≥ 160 mmHg or diastolic blood pressure is ≥ 100 mmHg at screening)
• Patients or patient's immediate family members (parents, siblings, offspring) with a history of malignant tumors within the last 5 years prior to the screening date, excluding basal cell carcinoma or squamous cell carcinoma that occurs on the skin (if it is determined that there is no possibility of relapse after resection), or with a family history of familial adenomatous polyposis (FAP) or hereditary nonpolyposis colorectal cancer (HNPCC)
• Patients who have not completed a national cancer screening program applicable to their sex and age (if it cannot be confirmed that the relevant test was received at a national cancer screening center or a recognized screening center)
• However, if it is confirmed that the relevant test was received at a national cancer screening center or a recognized screening center during the screening period, and that the results were within normal range, the patients may participate in the clinical study.
• Females: For females ≥ 40 years of age, normal range findings must be confirmed in a mammogram within 2 years. For females ≥ 20 years of age, normal range findings must be confirmed in a Pap smear within 2 years.
• Patients diagnosed with active pulmonary tuberculosis

Sponsors & Collaborators

• Helixmith Co., Ltd.: lead

Study Sites (1)

Samsung Medical Center, Seoul National University Hospital

Seoul, 06351, South Korea

Location

Results Point of Contact

• Title: Jinsub Lee, PhD
• Organization: Helixmith, Co., Ltd.

jinsub.lee@helixmith.com

+82-10-8256-0439

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single center, repeat dose, open label, phase I/IIA clinical study

Study Record Dates

• First Submitted: March 31, 2022
• First Posted: May 4, 2022
• Study Start: September 21, 2020
• Primary Completion: September 9, 2021
• Study Completion: February 22, 2022
• Last Updated: October 6, 2025
• Results First Posted: May 29, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)