Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A (NCT05333406) | Clinical Trial Compass
CompletedPhase 1
Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A
South Korea9 participantsStarted 2022-02-22
Plain-language summary
Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Charcot-Marie-Tooth disease (CMT) type 1A
Who can participate
Age range
19 Years – 75 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 and females aged 19 to 75 years old
. Those diagnosed with CMT type 1A by a genetic test
. Those whose CMTNS-v2 score is more than 2 and 20 or fewer points, and the severity of the disease is mild to moderate
. Those who have dorsiflexion muscle weakness
. Those who can comply with the requirements for clinical trials
. For women of childbearing potential, those who have a negative urine pregnancy test at screening
. Those who use a medically acceptable method of contraception until clinical trial visit 7 (short-term follow-up visit, 16 weeks): hormonal contraception, intrauterine device (IUD), intrauterine system (IUS), vasectomy, tubal ligation, or double barrier method using a cervical cap or a diaphragm with a male condom.
. Those who voluntarily agree to participate in this study and sign an IRB-approved consent form after being informed about the characteristics of this clinical trial prior to all screening tests
Exclusion criteria
. Those with other neuromuscular diseases that the investigator judges cannot participate in the clinical trial
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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
Number of participants of any Adverse Events (AEs)/Serious Adverse Events (SAEs) related investigational product
Timeframe: Week 16 after treatment
2
Determination of Dose-limiting toxicity (DLT) levels of EN001
Timeframe: Up to Week 4 after dosing on Day 0
3
Determination of Maximum tolerated dose (MTD) levels of EN001
Timeframe: Up to Week 4 after dosing on Day 0
4
Number of participants with Vital Signs abnormalities
Timeframe: From screening up to Week 16
5
Number of participants with clinically significant abnormalities of Physical Examinations
Timeframe: From screening up to Week 16
6
Number of participants with abnormalities of Laboratory Parameters
Timeframe: From screening up to Week 16
7
Number of participants with 12-lead Electrocardiography (ECG) abnormalities
. Patients diagnosed with type 1 or type 2 diabetes
. Those with a history of stroke or cerebral ischemic attack within 12 months of screening
. Those with a history of coronary artery diseases such as myocardial infarction or unstable angina within 12 months of screening
. Those who have undergone orthopedic surgery on the lower extremities (bone and ligament correction, artificial joint insertion, osteotomy, arthroscopic surgery) within 6 months of screening
. Those who have ankle contractures or have surgery that may affect muscle strength assessment
. Those who have experience with stem cell therapy or gene therapy before screening
. Those who have participated in clinical trials for chemical synthetic drugs before screening (except when 5 times the half-life has passed)
Timeframe: From screening to baseline on Day 1 (Predose to end of infusion and 90 min after completion of infusion)