Status:
COMPLETED
Evaluate the Safety and Potential Efficacy of Human Wharton's Jelly-derived Mesenchymal Stem Cells With Charcot-Marie-Tooth Disease Type 1E
Lead Sponsor:
Samsung Medical Center
Conditions:
Charcot-Marie-Tooth Disease, Type 1
Eligibility:
All Genders
19+ years
Phase:
NA
Brief Summary
Charcot-Marie-Tooth disease (CMT) is a hereditary peripheral nerve disease that causes causes muscle atrophy, muscle weakness, sensory loss, balance disorder, gait disorder, blindness, hearing disorde...
Eligibility Criteria
Inclusion
- Those who voluntarily agreed to participate in this clinical study
- Men and women over 19 years of age as of the date of written consent
- 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
- 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
- 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.
- Medical history and surgical history
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- 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
- 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
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- A person who participated in another clinical trial and administered/applied an investigational drug or medical device within 4 weeks before screening
- Those who administered/applied immunosuppressants, chemotherapy, radiation therapy, etc. within 12 weeks before screening
- Those who administered stem cell therapy or gene therapy within 240 weeks before screening
- 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.
Key Trial Info
Start Date :
May 23 2024
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 28 2024
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT06218134
Start Date
May 23 2024
End Date
October 28 2024
Last Update
December 24 2025
Active Locations (1)
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1
SMC
Seoul, Gangnam-gu, South Korea, 06351