Last updated: March 18, 2024
Sponsor: ENCell
Overall Status: Active - Recruiting
Phase
1
Condition
Neuropathy
Peripheral Neuropathy
Treatment
EN001
Clinical Study ID
NCT06328712
EN001_MIRACLE1
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 more and 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, tuballigation, 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 also acceptable.However, periodic abstinence (e.g. Karenda method, ovulation method,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 mmHg ordiastolic blood pressure over 110 mmHg)
- Subjects with other clinically significant diseases, including significant heart,lung, liver, kidney, hematological, immunological or behavioral diseases ormalignant 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 the HBVDNA test result is negative. (If anti-HCV positive) However, registration ispossible if the HCV RNA test result is negative.
- Those who have ankle contracture or have undergone surgery that may affect musclestrength 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 weeks beforescreening
- Those with a history of coronary artery disease, such as myocardial infarction orincomplete 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 their lives
- 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 to thejudgment of the investigator
Study Design
Total Participants: 12
Treatment Group(s): 1
Primary Treatment: EN001
Phase: 1
Study Start date:
March 01, 2024
Estimated Completion Date:
October 31, 2024
Study Description
Connect with a study center
Samsung Medical Center
Seoul,
Korea, Republic ofActive - Recruiting


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