Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A

NCT06328712 | Completed Phase 1

• 1 other identifier: EN001_MIRACLE1
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

An Open, Dose-escalation, Phase 1b Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients with Charcot-Marie-Tooth Disease type 1A (CMT1A)

Conditions

Charcot-Marie-Tooth Disease Type 1A

Outcome Measures

Primary Outcomes (1)

• Dose limiting toxicity (DLT) and adverse drug reactions related to discontinuation of investigational product administration

  Present the frequency and percentage of dose-limiting toxicity (DLT) occurrence across dose cohorts, along with detailed information on the types of DLTs. Adverse events related to discontinuation of clinical investigational drug administration, discontinuation of clinical investigational drug administration Regarding related adverse drug reactions, the number of subjects in each cohort, incidence rate (%), and Two-sided 95% confidence intervals and number of occurrences are presented.

  Up to 8 weeks

Secondary Outcomes (19)

• CMTNSv2 score change

  At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)

• CMT examination score change

  At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)

• Rasch-modified CMTNSv2 score change

  At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)

• Rasch-modified CMTES score change

  At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)

• FDS score change

  At 4, 8, 18, and 24 weeks compared to baseline (Visit 2)

Study Arms (2)

Cohort 1

ACTIVE COMPARATOR

EN001 1.25×10\^6 cells/kg

Drug: EN001

Cohort 2

ACTIVE COMPARATOR

EN001 2.5×10\^6 cells/kg

Drug: EN001

Interventions

EN001 DRUG

* Cohort 1: EN001 1.25×10\^6 cells/kg administered intravenously (IV) 2 times at 4 week intervals. * Cohort 2: EN001 2.5×10\^6 cells/kg administered intravenously (IV) 2 times at 4 week intervals.

Also known as: EN001(Allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells(WJ-MSCs) )

Cohort 1; Cohort 2

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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

Sponsors & Collaborators

• ENCell: lead

Study Sites (1)

Samsung Medical Center

Seoul, South Korea

Location

MeSH Terms

Conditions

Charcot-Marie-Tooth Disease

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 19, 2024
• First Posted: March 25, 2024
• Study Start: May 30, 2024
• Primary Completion: November 1, 2025
• Study Completion: November 1, 2025
• Last Updated: December 23, 2025

Record last verified: 2025-12

Locations

KR (1)