RecruitingPhase 1Phase 2NCT06328712
Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A(CMT1A) (Phase 1b: Open-label, Dose-escalation, Single-center; Phase 2a: Randomized, Double-blind, Placebo-controlled, Multicenter)
A Phase 1b/2a Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A(CMT1A) (Phase 1b: Open-label, Dose-escalation, Single-center; Phase 2a: Randomized, Double-blind, Placebo-controlled, Multicenter)
Sponsor
ENCell
Enrollment
27 participants
Start Date
May 30, 2024
Study Type
INTERVENTIONAL
Conditions
Summary
A Phase 1b/2a Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients with Charcot-Marie-Tooth Disease type 1A(CMT1A) (Phase 1b: Open-label, Dose-escalation, Single-center; Phase 2a: Randomized, Double-blind, Placebo-controlled, Multicenter)
Eligibility
Min Age: 19 Years
Inclusion Criteria14
- 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 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 Criteria33
- Those with the following comorbidities confirmed at the time of screening
- 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.
- 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 or diastolic blood pressure over 110 mmHg)
- 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
- 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.
- Those who have ankle contracture or have undergone surgery that may affect muscle 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 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
- 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)
- Drugs and therapies prohibited from concurrent use
- Those who participated in another clinical trial and administered/applied clinical trial drugs/medical devices within 4 weeks before screening
- Those who administered/applied immunosuppressants, chemotherapy, radiation therapy, 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 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
- Persons with hypersensitivity to the components of clinical investigational products
- Those who have had metal substances (heart pacemaker, nerve stimulator, cochlear implant, 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 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.
- Those who are deemed inappropriate to participate in clinical trials according to the judgment of the investigator
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Interventions
DRUGEN001
* Cohort 1: EN001 Low dose administered intravenously (IV) 2 times at 4 week intervals. * Cohort 2: EN001 High dose administered intravenously (IV) 2 times at 4 week intervals.
DRUGEN001 Placebo
EN001 Placebo administered intravenously (IV) 2 times at 4 week intervals.
Locations(3)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06328712