Phase
Condition
Neuropathy
Peripheral Neuropathy
Treatment
EN001
Clinical Study ID
Ages > 19 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
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 moreand 20 or less.
- Those experiencing muscle weakness due to foot dorsiflexion impairment.
- Women and men of childbearing potential who have agreed to use the appropriatecontraceptive method(s) outlined in the protocol during the clinical trial period.
- Appropriate contraception is defined as follows and is achieved by applying oneor 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 contraceptivemethods [hormonal contraceptives (oral contraceptives, subcutaneouscontraceptives (Implanon, etc.), long-acting contraceptive injections,emergency contraceptive pills), implantation of an intrauterine device orintrauterine system (Loop, Mirena), Infertility procedures (vasectomy,tubal ligation, etc.)]
- Abstinence: Absolute abstinence. If, in the examiner's judgment, thesubject's age, occupation, lifestyle, or sexual orientation warrantscontraception, strict abstinence from sexual intercourse is alsoacceptable. However, periodic abstinence (e.g. Karenda method, ovulationmethod, symptomatic temperature method), abstinence, and external vaginalejaculation are not recognized as appropriate contraceptive methods.
Exclusion
Exclusion Criteria:
Those with the following comorbidities confirmed at the time of screening
Subjects with neuromuscular diseases other than CMT1A or neuropathy- inducingfactors (uremia) that may affect the safety and efficacy evaluation of thisclinical 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 mmHgor diastolic blood pressure over 110 mmHg)
Subjects with other clinically significant diseases, including significantheart, lung, liver, kidney, hematological, immunological or behavioral diseasesor malignant tumors, according to the investigator's judgment
Individuals who display the specified test abnormalities in laboratory tests atthe 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) ispositive (If anti-HBc positive) However, registration is possible if theHBV 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 affectmuscle 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 lowerextremities within 24 weeks before screening
Those with a history of stroke or cerebral ischemic attack within 48 weeksbefore screening
Those with a history of coronary artery disease, such as myocardial infarctionor 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 theskin)
Drugs and therapies prohibited from concurrent use
Those who participated in another clinical trial and administered/appliedclinical trial drugs/medical devices within 4 weeks before screening
Those who administered/applied immunosuppressants, chemotherapy, radiationtherapy, etc. within 12 weeks before screening
Persons who have administered cell therapy or gene therapy throughout theirlives
Persons who have administered neurotoxic drugs that can accelerate peripheralnerve 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
Persons with hypersensitivity to the components of clinical investigational products
Those who have had metal substances (heart pacemaker, nerve stimulator, cochlearimplant, 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 othersignificant mental disorder) or a history of drug and alcohol abuse that may affectthe clinical trial, according to the judgment of the investigator.
Those who are deemed inappropriate to participate in clinical trials according tothe judgment of the investigator
Study Design
Study Description
Connect with a study center
Samsung Medical Center
Seoul,
Korea, Republic ofSite Not Available

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