The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subt… (NCT05361031) | Clinical Trial Compass
CompletedPhase 1/2
The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)
South Korea12 participantsStarted 2020-09-21
Plain-language summary
To assess the safety and tolerability of the investigational product Engensis (VM202) injected in the weakened lower limb muscles of CMT1A patients
Who can participate
Age range
19 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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
Exclusion criteria
. 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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this is a Phase 1/2 trial focused on safety, what does that mean for how much we currently know about whether Engensis actually helps slow or reverse CMT1A symptoms — and is that enough to consider it?
2The trial used intramuscular injections of Engensis directly into the muscles — can you walk me through what that procedure involves and what kinds of side effects or risks were being monitored?
3Now that this trial is completed, have any results been published or shared that you've seen, and do they suggest the treatment was well-tolerated in CMT1A patients?
4Given that this studied a specific subtype — CMT1A — how important is it that my exact genetic subtype is confirmed before we even discuss whether findings from this trial would be relevant to my situation?
5Are there any follow-up trials or next steps planned based on this study that might be worth watching, or would standard supportive care still be the more established path for me right now?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Safety of Intramuscular (IM) Injections of Engensis in Participants
. 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