A Phase 1/2 interventional study of EN001 and EN001 Placebo in Charcot-Marie-Tooth Disease Type 1A, sponsored by ENCell. Recruiting at 3 sites in South Korea. Open to participants aged 19 Years and older. Per ClinicalTrials.gov, last updated 2026-05-26.
Sponsored by ENCell · Phase 1/2, Interventional, and Treatment
A Phase 1b/2a Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients with Charcot-Marie-Tooth Disease type 1A(CMT1A) (Phase 1b: Open-label, Dose-escalation, Single-center; Phase 2a: Randomized, Double-blind, Placebo-controlled, Multicenter)
This clinical trial consists of two stages (Phase 1b and Phase 2a). Phase 1b is designed with a 3+3 dose escalation design to evaluate the safety, including tolerability, of EN001 and explore efficacy. Phase 2a will evaluate the efficacy and safety of EN001 in comparison with placebo.
-Phase 1b The study was designed using the traditional 3+3 dose escalation method to confirm the maximum tolerated dose (MTD) and determine the recommended phase 2 dose (RP2D).
Dose increase is carried out until the maximum tolerated dose (MTD) is confirmed at the high dose (Cohort 2), which is the maximum planned dose (MPD), or at a lower dose. The maximum tolerated dose (MTD) is defined as the highest dose at which the incidence of dose limiting toxicity (DLT) is lower than 33%. To determine the maximum tolerated dose (MTD), 3-6 test subjects from each dose cohort are enrolled and EN001 is administered twice at 4-week intervals, and dose-limiting toxicity (DLT) is evaluated until 4 weeks (visit 6).
The safety review committee (SRC) is comprised of the principal investigator, sponsor, etc. as members, and EN001 confirmed by the end of each cohort (end of dose-limiting toxicity (DLT) evaluation of the last dosed subject in the cohort). Safety data are comprehensively reviewed to determine all matters related to dose, such as increase or decrease in dose, and finally the recommended phase 2 dose (RP2D) is determined.
-Phase 2a The Phase 2a is a randomized, double-blind, placebo-controlled clinical trial. Eligible subjects will be randomly assigned in a 1:1:1 ratio to Study Group 1 (EN001 Low dose), Study Group 2 (EN001 High dose), or the placebo control group. The efficacy and safety of EN001 will be evaluated in comparison with placebo.
In addition, test subjects participating in phase 1b/2a will be followed up for safety and effectiveness for 5 years from the time of last EN001 administration according to the long-term follow-up protocol.
129 studies on the registry are indexed under Charcot-Marie-Tooth Disease; 38 are open to participants now.
This study's planned enrollment of 27 is below the median of 47 across 73 interventional studies indexed under Charcot-Marie-Tooth Disease.
Browse Charcot-Marie-Tooth Disease studies →ENCell is the lead sponsor of 5 studies on the registry; 3 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Individuals who meet all of the following genetic and clinical diagnostic criteria:
Clinical diagnosis:
Women and men of childbearing potential who have agreed to use the appropriate contraceptive method(s) outlined in the protocol during the clinical trial period.
Appropriate contraception is defined as follows and is achieved by applying one or more methods of contraception.
Exclusion Criteria:
Those with the following comorbidities confirmed at the time of screening
Individuals who display the specified test abnormalities in laboratory tests at the time of screening:
Medical history and surgical history
Drugs and therapies prohibited from concurrent use
Persons who have administered neurotoxic drugs that can accelerate peripheral nerve damage ① Within 1 week of Screening
Dichloroacetate
② Within 55 weeks of Screening
Phase 1b - Low dose
Drug: EN001
EN001 High dose
Drug: EN001
EN001 Low dose
Drug: EN001
EN001 High dose
Drug: EN001
EN001 Placebo
Drug: EN001 Placebo
* Cohort 1: EN001 Low dose administered intravenously (IV) 2 times at 4 week intervals. * Cohort 2: EN001 High dose administered intravenously (IV) 2 times at 4 week intervals.
Also known as: EN001(Allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells(WJ-MSCs) )
EN001 Placebo administered intravenously (IV) 2 times at 4 week intervals.
[Phase 1b] Dose limiting toxicity (DLT) and adverse drug reactions related to discontinuation of investigational product administration
Present the frequency and percentage of dose-limiting toxicity (DLT) occurrence across dose cohorts, along with detailed information on the types of DLTs. Adverse events related to discontinuation of clinical investigational drug administration, discontinuation of clinical investigational drug administration Regarding related adverse drug reactions, the number of subjects in each cohort, incidence rate (%), and Two-sided 95% confidence intervals and number of occurrences are presented.
Time frame: Up to 8 weeks
[Phase 2a] Change from baseline in the CMT Neuropathy Score (CMTNSv2) at Week 24.
For all efficacy outcome measures, descriptive statistics (number of subjects, mean, standard deviation, median, minimum, and maximum) will be presented by treatment group and assessment time point, along with two-sided 95% confidence intervals.
Time frame: at Week 24
[Phase1b, Phase2a] CMTNSv2 score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] CMT examination score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Rasch-modified CMTNSv2 score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Rasch-modified CMTES score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] FDS score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] ONLS score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] 10MWT score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase 1b] Change in grade of fatty infiltration in the proximal lower extremities (According to Goutallier classification scale)
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Change in Motor Nerve Conduction Velocity (Unit: m/s)
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Change in Compound Muscle Action Potential (Unit: ㎷)
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Change in Sensory Nerve Action Potential (Unit: ㎶)
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Change in Sensory Nerve Conduction Velocity (Unit: m/s)
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
SF-36v2 score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Time frame: At 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Adverse Event
By cohort that Number of subjects, incidence rate (%), confidence interval (two-sided 95%), presents the number of occurrences. Additional, The pre-treatment adverse event that occurred before administration of clinical trial drugs is presented in detail.
Time frame: Up to 24weeks. However, adverse drug reactions that persist at the end of the clinical trial will be followed up until the possible adverse reactions are resolved or it is determined that further follow-up is not meaningful.
[Phase1b, Phase2a] Vital sign
Number of participants with clinically significant abnomalities in vital signs after EN001 administration. Vital Signs include blood pressure (mmHg), pulse (times/minute), respiratory rate (times/minute), and body temperature (℃) and will be assessed. By cohort and visit, present continuous variables with subject count, mean, standard deviation, median, minimum, and maximum. For categorical variables, provide shift table.
Time frame: Up to 24 weeks
[Phase1b, Phase2a] Laboratory examination
Number of participants with clinically significant abnormalities in Laboratory parameters after EN001 administration. Hematological tests, Blood chemical tests, Blood coagulation test, Urine test, Serum virus test. It is conducted through blood and urine collection, and the PI checks whether the test results are normal, abnormal, and clinically significant.
Time frame: Up to 24 week. Serum virus testing is performed only during screening. At Baseline and Week 4, hematological tests, blood chemical tests, TCO2, Mg, and blood coagulation tests are performed before and within 4 hours after administration of the IP.
[Phase1b, Phase2a] electrocardiography
Number of participants with clinically significant abnormalities in electrocardiography after EN001 administration. Based on the PR Interval, QRS Duration, QTc Interval and QTcF Interval, PI checks whether the test results are normal, abnormal, and clinically significant. by cohort and visit, present continuous variables with subject count, mean, standard deviation, median, minimum, and maximum. For categorical variables, provide shift table.
Time frame: At Baseline, Week 4, Week 8, Week 12, Week 18, Week 24
[Phase1b, Phase2a] X-ray
by cohort and visit, present continuous variables with subject count, mean, standard deviation, median, minimum, and maximum. For categorical variables, provide shift table.
Time frame: Before administration of investigational product at Baseline and Week 4 and within 4 hours after completion of administration
[Phase1b, Phase2a] Physical Examinations
Number of participants with clinically significant abnormalities in Physical Examinations after EN001 administration. Based on general appearance, head, ears/eyes/nose/throat, cardiovascular, respiratory, abdomen, skin, lymph nodes, extremities, musculoskeletal and neurologic, PI checks whether the test results are normal, abnormal, and clinically significant. By cohort and visit, present continuous variables with subject count, mean, standard deviation, median, minimum, and maximum. For categorical variables, provide shift table.
Time frame: Up to 24 weeks
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