Charcot-Marie-Tooth Disease Type 1APhase 1/2NCT06328712
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)
A Phase 1/2 study testing EN001 in people with Charcot-Marie-Tooth Disease Type 1A, run by ENCell at 3 sites in South Korea.
Trial summary
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) Read more about this study
- Who is it for?
- Ages 19 years and older
- Conditions the study is for
- Charcot-Marie-Tooth Disease Type 1A
- What is being tested?
- EN001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 3 sitesSouth Korea
- Study dates
- 30 May 2024to 31 Dec 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Individuals who have voluntarily agreed to participate in this clinical trial.
- Men and women aged 19 years or older at the time of providing written consent.
- Individuals who meet all of the following genetic and clinical diagnostic criteria:
- Genetic diagnosis: CMT1A type
- Clinical diagnosis:
- Those with a CMT Neuropathy Score version 2 (CMTNSv2) between 2 or more and 20 or less.
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- Those experiencing muscle weakness due to foot dorsiflexion impairment.
- 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.
- Hormonal contraceptives
- Implantation of an intrauterine device or intrauterine system
- Sterilization procedures (vasectomy, tubal ligation, etc.)
- Double contraceptive method: male condom along with other contraceptive methods [hormonal contraceptives (oral contraceptives, subcutaneous contraceptives (Implanon, etc.), long-acting contraceptive injections, emergency contraceptive pills), implantation of an intrauterine device or intrauterine system (Loop, Mirena), Infertility procedures (vasectomy, tubal ligation, etc.)]
- Abstinence: Absolute abstinence. If, in the examiner's judgment, the subject's age, occupation, lifestyle, or sexual orientation warrants contraception, strict abstinence from sexual intercourse is also acceptable. However, periodic abstinence (e.g. Karenda method, ovulation method, symptomatic temperature method), abstinence, and external vaginal ejaculation are not recognized as appropriate contraceptive methods.
Who cannot join
- Those with the following comorbidities confirmed at the time of screening
- Subjects with neuromuscular diseases other than CMT1A or neuropathy- inducing factors (uremia) that may affect the safety and efficacy evaluation of this clinical trial, according to the judgment of the investigator.
- Individuals diagnosed with type 1 or type 2 diabetes
- Individuals diagnosed with active pulmonary tuberculosis
- Patients with uncontrolled hypertension (systolic blood pressure over 180 mmHg or diastolic blood pressure over 110 mmHg)
- Subjects with other clinically significant diseases, including significant heart, lung, liver, kidney, hematological, immunological or behavioral diseases or malignant tumors, according to the investigator's judgment
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- Individuals who display the specified test abnormalities in laboratory tests at the time of screening:
- AST or ALT > 3 x ULN
- Total bilirubin> 1.5 x ULN
- Serum creatinine > 1.5 x ULN
- Any one of the serum virus tests (HBsAg, anti-HBc, anti-HCV, HIV Ag/Ab) is positive (If anti-HBc positive) However, registration is possible if the HBV DNA test result is negative. (If anti-HCV positive) However, registration is possible if the HCV RNA test result is negative.
- Those who have ankle contracture or have undergone surgery that may affect muscle strength measurement tests
- Medical history and surgical history
- Those who have undergone orthopedic surgery (bone or ligament correction, artificial joint implantation, osteotomy, arthroscopic surgery) on the lower extremities within 24 weeks before screening
- Those with a history of stroke or cerebral ischemic attack within 48 weeks before screening
- Those with a history of coronary artery disease, such as myocardial infarction or incomplete angina, within 48 weeks before screening
- Those with a history of malignant tumor within 240 weeks before screening (excluding basal cell carcinoma or squamous cell carcinoma that occurs on the skin)
- Drugs and therapies prohibited from concurrent use
- Those who participated in another clinical trial and administered/applied clinical trial drugs/medical devices within 4 weeks before screening
- Those who administered/applied immunosuppressants, chemotherapy, radiation therapy, etc. within 12 weeks before screening
- Persons who have administered cell therapy or gene therapy throughout their lives
- Persons who have administered neurotoxic drugs that can accelerate peripheral nerve damage ① Within 1 week of Screening
- Anti-inflammatory agents or antibiotics: Colchicine, Nitrofurantoin
- Antiretroviral agents: Zalcitabine, Stavudine
- Dichloroacetate
- ② Within 55 weeks of Screening
- Antiarrhythmic agent: Amiodarone
- Antiparasitic agent: Suramin
- Persons with hypersensitivity to the components of clinical investigational products
- Those who have had metal substances (heart pacemaker, nerve stimulator, cochlear implant, etc.) implanted in their body
- Pregnant, lactating, or planning to become pregnant during the clinical trial period
- Subjects with a psychiatric disorder (anxiety disorder, claustrophobia, or other significant mental disorder) or a history of drug and alcohol abuse that may affect the clinical trial, according to the judgment of the investigator.
- Those who are deemed inappropriate to participate in clinical trials according to the judgment of the investigator
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
South Korea3 sites, 3 recruiting
- Recruiting
Dongguk University Gyeongju Hospital
Gyeongju
Jin-Mo Park
- Recruiting
Kyung Hee University Hospital at Gangdong
Seoul
Sang Beom Kim
- Recruiting
Samsung Medical Center
Seoul
Byung-Ok Choi
About this study
Official title: 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)
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)
Read the detailed description
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.
- Conditions
- Charcot-Marie-Tooth Disease Type 1A
- Being tested
- EN001, EN001 Placebo
- Keywords
- EN001, CMT1A, ENCell, MSC, CMT
- Medical subject headings
- Charcot-Marie-Tooth Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EN001Drug
Also called EN001(Allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells(WJ-MSCs) )
- 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.
EN001 PlaceboDrug
EN001 Placebo administered intravenously (IV) 2 times at 4 week intervals.
| Study group | Role | What they receive |
|---|---|---|
| Phase1b - Cohort 1 | Active comparator | Phase 1b - Low dose Receives: Drug: EN001 |
| Phase 1b - Cohort 2 | Active comparator | EN001 High dose Receives: Drug: EN001 |
| Phase 2a - Cohort 1 | Active comparator | EN001 Low dose Receives: Drug: EN001 |
| Phase 2a - Cohort 2 | Active comparator | EN001 High dose Receives: Drug: EN001 |
| Phase 2a - Placebo | Placebo comparator | EN001 Placebo Receives: Drug: EN001 Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
[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.
Measured: 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.
Measured: at Week 24
Secondary outcomes (19)
[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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2024
- Study start30 May 2024
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Charcot-Marie-Tooth Disease Type 1AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Phase 1b Design : 3+3 Dose-escalation, Open label Phase 2a Design : Double-blind
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 27 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ENCell
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06328712
- Sponsor's protocol ID
- EN001_MIRACLE1
- Status last verified
- May 2026
- First submitted
- 19 Feb 2024
- Last updated
- 26 May 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT06328712
- Acronym / sponsor ID
- EN001_MIRACLE1
- Conditions
- Charcot-Marie-Tooth Disease Type 1A
- Keywords
- EN001, CMT1A, ENCell, MSC, CMT
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 27 participants (estimated)
- Lead sponsor
- ENCellIndustry (pharma, biotech, devices)
- Study start
- 30 May 2024 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 25 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
- Model description
- Phase 1b Design : 3+3 Dose-escalation, Open label Phase 2a Design : Double-blind
| Arm | Type | Receives |
|---|---|---|
| Phase1b - Cohort 1 | Active comparator | Drug: EN001Phase 1b - Low dose |
| Phase 1b - Cohort 2 | Active comparator | Drug: EN001EN001 High dose |
| Phase 2a - Cohort 1 | Active comparator | Drug: EN001EN001 Low dose |
| Phase 2a - Cohort 2 | Active comparator | Drug: EN001EN001 High dose |
| Phase 2a - Placebo | Placebo comparator | Drug: EN001 PlaceboEN001 Placebo |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Individuals who have voluntarily agreed to participate in this clinical trial.
- Men and women aged 19 years or older at the time of providing written consent.
- Individuals who meet all of the following genetic and clinical diagnostic criteria:
- 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.
Key exclusion criteria (33 listed)
- Those with the following comorbidities confirmed at the time of screening
- Medical history and surgical history
- Drugs and therapies prohibited from concurrent use
- ② Within 55 weeks of Screening
- Persons with hypersensitivity to the components of clinical investigational products
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EN001Also: EN001(Allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells(WJ-MSCs) )* 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. | Drug | Phase 1b - Cohort 2, Phase 2a - Cohort 1, Phase 2a - Cohort 2, Phase1b - Cohort 1 |
| EN001 PlaceboEN001 Placebo administered intravenously (IV) 2 times at 4 week intervals. | Drug | Phase 2a - Placebo |
Primary outcome
[Phase 1b] Dose limiting toxicity (DLT) and adverse drug reactions related to discontinuation of investigational product administration
Time frame: Up to 8 weeks
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.
[Phase 2a] Change from baseline in the CMT Neuropathy Score (CMTNSv2) at Week 24.
Time frame: 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.
19 secondary outcomes
[Phase1b, Phase2a] CMTNSv2 score change
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] CMT examination score change
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Rasch-modified CMTNSv2 score change
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Rasch-modified CMTES score change
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] FDS score change
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] ONLS score change
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] 10MWT score change
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)
Time frame: At 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Change in Motor Nerve Conduction Velocity (Unit: m/s)
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Change in Compound Muscle Action Potential (Unit: ㎷)
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Change in Sensory Nerve Action Potential (Unit: ㎶)
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)
Time frame: At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)
SF-36v2 score change
Time frame: At 24 weeks compared to baseline (Visit 2)
[Phase1b, Phase2a] Adverse Event
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
Time frame: Up to 24 weeks
[Phase1b, Phase2a] Laboratory examination
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
Time frame: At Baseline, Week 4, Week 8, Week 12, Week 18, Week 24
[Phase1b, Phase2a] X-ray
Time frame: Before administration of investigational product at Baseline and Week 4 and within 4 hours after completion of administration
[Phase1b, Phase2a] Physical Examinations
Time frame: Up to 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 May 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing EN001 in people with Charcot-Marie-Tooth Disease Type 1A, run by ENCell at 3 sites in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 33 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact ENCell (+82-2-6205-8054, encell@encellinc.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
[Phase 1b] Dose limiting toxicity (DLT) and adverse drug reactions related to discontinuation of investigational product administration (time frame: Up to 8 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 May 2026.