Trial Outcomes & Findings for The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A) (NCT NCT05361031)

Race and Ethnicity were not collected from any participant.

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.

Charcot-Marie-Tooth Neuropathy Score version 2 is a measurement tool for evaluating the severity of disease. The scoring system is the total neuropathy score, by assessing the symptoms of Charcot-Marie-Tooth patients due to sensory impairment. Measurements were performed in three items for the symptoms of disease, four items for the signs, and two items for neurophysiological testing, a total of nine separate test items. The Total Severity Score is the summed scores of all subscores and has a total minimum score of 0 and a maximum score of 36. The Total Severity Score Scale of the disease is classified according to the Total Score as either mild (0 to 10), moderate (11 to 20), or severe (20 to 36). The Change from Baseline Total Severity Score is the difference between the Day 0 and the Day 270 scores. A negative difference in the Total Severity Score Change from Baseline to Day 270, indicates an improvement of the treated groups' total severity level.

Change from Baseline (Day 0) in Functional Disability Scale at Days 90, 180, and 270; as assessed using the Functional Disability Scale 0 - 8 points, where normal is 0 point, best case, and if determined as bedridden, it is evaluated as 8 points, worst case. 0=normal; 1=cramps and fatigability; 2=inability to run; 3=possible unaided; 4=with cane; 5=with crutch; 6=with walker; 7=wheelchair; 8=bedridden. Changes in severity of disease: Functional Disability Scale - Intent-to-Treat population.

Change from baseline (Day 0) in the overall neuropathy limitation score leg scale at Days 90, 180, and 270. The Overall Neuropathy Limitation Score is scored from 0 to 7. The worst score, 7 points, is defined as restricted to wheelchair, or bed most of the day, unable to make any purposeful movements of the leg.

This test measures the time in seconds required for a subject to walk 10 meters through the 10-Meter Walking Test version 2.2. Changes in the Mean Time in seconds for lower limb function assessment: 10 Meter Walk Test - Intent to Treat population, from Day 0 to Days 90, 180, and 270. The larger the change in time (Seconds) from Day 0 to Visit date indicates a worsening of the patients walking ability.

Changes from baseline (Day 0) in the fat content of lower limb muscles at Day 270. The muscles of lower limbs were imaged with Magnetic Resonance Imaging leg scan, and the degree of fatty infiltration of the leg muscles injected with the investigational product was measured and valuated as fat content value (percent) at one level for each muscle. The six MRI imaging sites to be measured were as follows: peroneus longus left/right, gastrocnemius left/right, and tibialis anterior left/right.

Change from baseline in Nerve Conduction Velocity to Day 270, for evaluation of the presence of a disorder in a neurotransmission pathway, as assessed using the Nerve Conduction Study version 2.0 Nerve regeneration potential (Electroneurography): Nerve Conduction Velocity (measured in meters per second) - Intent-to-Treat population. Larger negative change from baseline values indicates a slowing of the Nerve Conduction Velocity which is a worsening of the electroconduction function of the specified nerve.

The presence of hepatocyte growth factor antibody generation by Engensis (VM202) assessed using anti-hepatocyte growth factor antibody test at Day 0 and Day 270