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

ENCell

Status and phase

Completed

Phase 1

Conditions

Charcot-Marie-Tooth Disease, Type IA

Treatments

Drug: EN001

Study type

Interventional

Funder types

Industry

Identifiers

NCT05333406

ENCell_2020_02

Details and patient eligibility

About

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

Full 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.

Enrollment

9 patients

Sex

All

Ages

19 to 75 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Males and females aged 19 to 75 years old
Those diagnosed with CMT type 1A by a genetic test
Those whose CMTNS-v2 score is more than 2 and 20 or fewer points, and the severity of the disease is mild to moderate
Those who have dorsiflexion muscle weakness
Those who can comply with the requirements for clinical trials
For women of childbearing potential, those who have a negative urine pregnancy test at screening
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.
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

Those with other neuromuscular diseases that the investigator judges cannot participate in the clinical trial
Patients diagnosed with type 1 or type 2 diabetes
Those with a history of stroke or cerebral ischemic attack within 12 months of screening
Those with a history of coronary artery diseases such as myocardial infarction or unstable angina within 12 months of screening
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
Those who have ankle contractures or have surgery that may affect muscle strength assessment
Those who have experience with stem cell therapy or gene therapy before screening
Those who have participated in clinical trials for chemical synthetic drugs before screening (except when 5 times the half-life has passed)
Patients with uncontrolled hypertension (If the systolic blood pressure is 180 mmHg or higher or the diastolic blood pressure is 110 mmHg or higher)
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
Those who diagnosed with active pulmonary tuberculosis
Immunosuppressed patients who are taking immunosuppressants, chemotherapy, radiation therapy, etc.
Mental illness patients
Those who are pregnant or lactating
Those with significant heart, lung, liver, kidney, hematological, immunological, behavioral disease, or other clinically significant diseases including malignant tumors
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
Those who do not have the will or ability to comply with clinical trial procedures at the discretion of the investigator