Please sign in to follow a disease.
Stem cells injected to fight rare nerve disease
NCT ID NCT06328712
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests a stem cell product called EN001 in 27 adults with Charcot-Marie-Tooth disease type 1A, a genetic nerve disorder that causes muscle weakness. The study has two parts: first, an open-label phase to check safety and find the right dose; second, a placebo-controlled phase to see if it improves nerve function. Participants receive two intravenous infusions four weeks apart.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- EN001 (donor stem cells from umbilical cord tissue)
- What this could lead to
- If it works, this could point toward a treatment that slows or improves nerve damage in CMT1A, potentially reducing disability.
- What could go wrong
- This is a very early, small trial (27 people) focused mainly on safety. The stem cell approach is experimental, and it may not improve symptoms or could cause side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
About 27 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
May 2024
- Expected to finish
-
Dec 2026
An estimate. End dates often move.
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
19 years and older
- Sex
-
Anyone
- Healthy volunteers
-
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. Individuals who have voluntarily agreed to participate in this clinical trial. 2. Men and women aged 19 years or older at the time of providing written consent. 3. Individuals who meet all of the following genetic and clinical diagnostic criteria: 1. Genetic diagnosis: CMT1A type 2. Clinical diagnosis: * Those with a CMT Neuropathy Score version 2 (CMTNSv2) between 2 or more and 20 or less. * Those experiencing muscle weakness due to foot dorsiflexion impairment. 4. 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. Exclusion Criteria: 1. Those with the following comorbidities confirmed at the time of screening 1. 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. 2. Individuals diagnosed with type 1 or type 2 diabetes 3. Individuals diagnosed with active pulmonary tuberculosis 4. Patients with uncontrolled hypertension (systolic blood pressure over 180 mmHg or diastolic blood pressure over 110 mmHg) 5. 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 6. 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. 7. Those who have ankle contracture or have undergone surgery that may affect muscle strength measurement tests 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. 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) 3. Drugs and therapies prohibited from concurrent use 1. Those who participated in another clinical trial and administered/applied clinical trial drugs/medical devices within 4 weeks before screening 2. Those who administered/applied immunosuppressants, chemotherapy, radiation therapy, etc. within 12 weeks before screening 3. Persons who have administered cell therapy or gene therapy throughout their lives 4. 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 4. Persons with hypersensitivity to the components of clinical investigational products 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 during the clinical trial period 7. 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. 8. Those who are deemed inappropriate to participate in clinical trials according to the judgment of the investigator
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Charcot-Marie-Tooth disease type 1A are added.
Genom att skicka in godkänner du våra Användarvillkor
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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The places running it
3 sites. The list below names each one and where it is.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
-
Dongguk University Gyeongju Hospital
RECRUITINGGyeongju, South Korea
-
Kyung Hee University Hospital at Gangdong
RECRUITINGSeoul, South Korea
-
Samsung Medical Center
RECRUITINGSeoul, South Korea
More trials for these conditions
Other studies related to the condition(s) this trial covers.