Experimental stem cells tested for rare nerve disease
NCT ID NCT06218134
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This completed early trial tested a single intravenous dose of EN001 stem cells in 3 adults with Charcot-Marie-Tooth disease type 1E, a severe hereditary nerve disorder with no approved treatment. The main goal was to check safety, including infusion reactions, while also looking for signs of benefit using a standard neuropathy scale. The study builds on prior safety data from 9 patients with a related CMT type.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- human Wharton's jelly-derived mesenchymal stem cells (EN001)
- What this could lead to
- If successful, this could point toward a treatment that slows or stabilizes the progression of severe CMT1E, improving quality of life.
- What could go wrong
- This was a very small, early-phase trial with only 3 participants, so results may not apply to all patients. The treatment may not show clear benefit, and long-term effects are unknown.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Not a phased trial
Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.
- Participants
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3 people
The number who actually took part.
- Started
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May 2024
- Finished
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Oct 2024
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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19 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Those who voluntarily agreed to participate in this clinical study 2. Men and women over 19 years of age as of the date of written consent 3. Those who meet all of the following genetic and clinical diagnostic criteria * Genetic diagnosis: CMT1E * Clinical diagnosis * CMTNS score of 2 or more * Those with muscle weakness due to foot dorsiflexion 4. Women or men of childbearing potential who agreed to use the appropriate contraceptive method suggested in the protocol during the clinical study 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.) Exclusion Criteria: 1. Those with the following comorbidities confirmed at the time of screening (1) Those who have a neuromuscular disease other than CMT1E or a neuropathy- causing factor (uremia) that may affect the safety and treatment effect of this clinical study, according to the judgment of the researcher. (2) Those diagnosed with type 1 or type 2 diabetes (3) Those diagnosed with active pulmonary tuberculosis (4) Patients with uncontrolled hypertension (systolic blood pressure over 180 mmHg or diastolic blood pressure over 110 mmHg) (5) Persons with other clinically significant diseases, including significant heart, lung, liver, kidney, hematological, immunological or behavioral diseases or malignant tumors, according to the judgment of the researcher. (6) Those who show the following test abnormalities in clinical laboratory tests at the time of screening * AST(spartate aminotransferase) or ALT(alanine aminotransferase) \> 3 x ULN(upper limit of normal) ②Total bilirubin \> 1.5 x ULN(upper limit of normal) * Serum creatinine \> 1.5 x ULN(upper limit of normal) * D-dimer \> 1.5 x ULN(upper limit of normal) ⑤ Serum virus test (HBsAg, anti-HBc, anti-HCV, Positive for any one of HIV Ag/Ab) \- (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. 2. Medical history and surgical history <!-- --> 1. 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 2. Those with a history of stroke or cerebral ischemic attack within 48 weeks before screening 3. Those with a history of coronary artery disease, such as myocardial infarction or incomplete angina, within 48 weeks before screening 4. Persons with a history of malignant tumor within 240 weeks prior to screening (excluding basal cell carcinoma or squamous cell carcinoma that occurs on the skin) 3) Drugs and therapies prohibited from concurrent use <!-- --> 1. A person who participated in another clinical trial and administered/applied an investigational drug or medical device within 4 weeks before screening 2. Those who administered/applied immunosuppressants, chemotherapy, radiation therapy, etc. within 12 weeks before screening 3. Those who administered stem cell therapy or gene therapy within 240 weeks before screening 4. Persons who have administered neurotoxic drugs that may accelerate peripheral nerve damage \- Platinum series: cisplatin, carboplatin, oxaliplatin \- Taxane series: paclitaxel, docetaxel \- Proteasome inhibitors: bortezomib, carfilzomib, ixazomib, etc. \- thalidomide and derivatives: thalidomide, lenalidomide, pomalidomide \- Vinca alkaloid series: vincristine, vinblastine, vindesine, vinorelbine \- Antiarrhythmic drug: amiodarone \- Anti-inflammatory and antibiotic: colchicine, nitrofurantoin * Antiretroviral drugs: zalcitabine, stavudine * Others: dichloroacetate, tacrolimus, suramin 4) Persons with hypersensitivity to the components of human cells (EN001) used in this clinical study 5) Those who have had metal substances (heart pacemaker, nerve stimulator, cochlear implant, etc.) implanted in their body 6) Pregnant, lactating, or planning to become pregnant while participating in clinical research 7) Persons with a psychiatric disorder (anxiety disorder, claustrophobia, or other significant mental disorder) or a history of drug and alcohol abuse that may affect clinical research, according to the judgment of the researcher. 8) A person who is deemed inappropriate to participate in clinical research according to the judgment of the researcher.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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SMC
Seoul, Gangnam-gu, 06351, South Korea
More trials for these conditions
Other studies related to the condition(s) this trial covers.