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

• Status: Recruiting
• Trial end date: 2027-08-19
• Target enrollment: 0
• Participant gender: All

Summary

Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Charcot-Marie-Tooth disease (CMT) type 1A

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: ENCell

Criteria

Inclusion Criteria:

1. Males and females aged 19 to 75 years old

2. Those diagnosed with CMT type 1A by a genetic test

3. Those whose CMTNS-v2 score is more than 2 and 20 or fewer points, and the severity of the disease is mild to moderate

4. Those who have dorsiflexion muscle weaknes

5. Those who can comply with the requirements for clinical trials

6. For women of childbearing potential, those who have a negative urine pregnancy test at screening

7. Those who use a medically acceptable method of contraception until clinical trial visit 7 (short-term follow-up visit, 16 weeks): hormonal contraception, intrauterine device (IUD), intrauterine system (IUS), vasectomy, tubal ligation, or double barrier method using a cervical cap or a diaphragm with a male condom.

8. Those who voluntarily agree to participate in this study and sign an IRB-approved consent form after being informed about the characteristics of this clinical trial prior to all screening tests

Exclusion Criteria:

1. Those with other neuromuscular diseases that the investigator judges cannot participate in the clinical trial

2. Patients diagnosed with type 1 or type 2 diabetes

3. Those with a history of stroke or cerebral ischemic attack within 12 months of screening

4. Those with a history of coronary artery diseases such as myocardial infarction or unstable angina within 12 months of screening

5. Those who have undergone orthopedic surgery on the lower extremities (bone and ligament correction, artificial joint insertion, osteotomy, arthroscopic surgery) within 6 months of screening

6. Those who have ankle contractures or have surgery that may affect muscle strength assessment

7. Those who have experience with stem cell therapy or gene therapy before screening

8. Those who have participated in clinical trials for chemical synthetic drugs before screening (except when 5 times the half-life has passed)

9. Patients with uncontrolled hypertension (If the systolic blood pressure is 180 mmHg or higher or the diastolic blood pressure is 110 mmHg or higher)

10. If there is a history of malignant tumors other than basal cell carcinoma or squamous cell carcinoma occurring in the skin within 5 years of screening

11. Those who diagnosed with active pulmonary tuberculosis

12. Immunosuppressed patients who are taking immunosuppressants, chemotherapy, radiation therapy, etc.

13. Mental illness patients

14. Those who are pregnant or lactating

15. Those with significant heart, lung, liver, kidney, hematological, immunological, behavioral disease, or other clinically significant diseases including malignant tumors

16. Those who have a previous or current medical condition that may adversely affect the safety of the subject, make it difficult to complete treatment or affect the evaluation of clinical trial results at the discretion of the investigator

17. Those who do not have the will or ability to comply with clinical trial procedures at the discretion of the investigator