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)
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)
• 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.