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

NCT ID: NCT05333406

Last Updated: 2023-02-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-02-22
• Study Completion Date: 2022-12-20

Brief 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

Detailed Description

It is the first in human (FIH), 3+3 design clinical trial to evaluate the safety and tolerability and determine the maximum tolerated dose (MTD) of EN001 (allogeneic umbilical cord-derived mesenchymal stem cells) in the treatment of Charcot-Marie-Tooth disease (CMT) type 1A.

Conditions

Charcot-Marie-Tooth Disease, Type IA

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Dose group A (Low dose)

Participants will receive EN001 intravenously (IV) once on Day 0.

Group Type: EXPERIMENTAL

EN001

Intervention Type: DRUG

EN001 intravenously (IV) in the treatment of Charcot-Marie-Tooth disease (CMT) type 1A Dosage for each group is as follows.

Dose group A (Low dose): 5.0x10^5 cells/kg

Dose group B (High dose)

Participants will receive EN001 intravenously (IV) once on Day 0.

Group Type: EXPERIMENTAL

EN001

Intervention Type: DRUG

EN001 intravenously (IV) in the treatment of Charcot-Marie-Tooth disease (CMT) type 1A Dosage for each group is as follows.

Dose group B (High dose): 2.5x10^6 cells/kg

Interventions

EN001

EN001 intravenously (IV) in the treatment of Charcot-Marie-Tooth disease (CMT) type 1A Dosage for each group is as follows.

Dose group A (Low dose): 5.0x10^5 cells/kg

Intervention Type: DRUG

EN001

EN001 intravenously (IV) in the treatment of Charcot-Marie-Tooth disease (CMT) type 1A Dosage for each group is as follows.

Dose group B (High dose): 2.5x10^6 cells/kg

Intervention Type: DRUG

Other Intervention Names

EN001 (allogeneic umbilical cord-derived mesenchymal stem cells); EN001 (allogeneic umbilical cord-derived mesenchymal stem cells)

Eligibility 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 weakness

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

• Minimum Eligible Age: 19 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

ENCell

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Samsung Medical Center

Seoul, , South Korea

Countries

South Korea

Other Identifiers

ENCell_2020_02

Identifier Type: -

Identifier Source: org_study_id