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)

Recruiting now · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Charcot-Marie-Tooth Disease Type 1A

In brief

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)

Key facts

Study ID: NCT06328712
Run by: ENCell
People needed: 27
Starts: 2024-05-30
Expected to finish: 2026-12-31
Last updated by the study team: 2026-05-26

Who can join

Age: 19 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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.

You may not qualify if…

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

Where it is running

Dongguk University Gyeongju Hospital — Gyeongju, South Korea (enrolling)
Kyung Hee University Hospital at Gangdong — Seoul, South Korea (enrolling)
Samsung Medical Center — Seoul, South Korea (enrolling)

Full record on ClinicalTrials.gov

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