Status:
COMPLETED
Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A
Lead Sponsor:
ENCell
Conditions:
Charcot-Marie-Tooth Disease, Type IA
Eligibility:
All Genders
19-75 years
Phase:
PHASE1
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...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
February 22 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 20 2022
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT05333406
Start Date
February 22 2022
End Date
December 20 2022
Last Update
February 17 2023
Active Locations (1)
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1
Samsung Medical Center
Seoul, South Korea