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Tests treatment safety and results for Charcot-Marie-Tooth Disease Type 1A
Official title Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A(CMT1A) (Phase 1b: Open-label, Dose-escalation, Single-center; Phase 2a: Randomized, Double-blind, Placebo-controlled, Multicenter)
ClinicalTrials.gov ID: NCT06328712
What this study is testing
What is EN001?
EN001 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for charcot-marie-tooth disease type 1a.
Also referred to as EN001(Allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells(WJ-MSCs) ).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- A Phase 1b/2a Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients with Charcot-Marie-Tooth Disease type 1A(CMT1A) (Phase 1b: Open-label, Dose-escalation, Single-center; Phase 2a: Randomized, Double-blind, Placebo-controlled, Multicenter)
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 19 and older
You may be able to join 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:
You likely can't join if
- Those with the following comorbidities confirmed at the time of screening
- people with neuromuscular diseases other than CMT1A or neuropathy- inducing factors (uremia) that may affect the safety and how well it works...
- 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)
- people with other clinically significant diseases, including significant heart, lung, liver, kidney, hematological, immunological or behavioral...
See the full eligibility criteria
- 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...
- 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...
- Those with the following comorbidities confirmed at the time of screening
- people with neuromuscular diseases other than CMT1A or neuropathy- inducing factors (uremia) that may affect the safety and how well it works evaluation of this clinical trial, according to the judgment of the...
- 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)
- people 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...
- 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
- Those with a history of coronary artery disease, such as myocardial infarction or incomplete angina, within 48 weeks before screening
- Those with a history of malignant tumor within 240 weeks before screening (excluding basal cell carcinoma or squamous cell carcinoma that occurs on the skin)
- Drugs and therapies prohibited from concurrent use
- Those who participated in another clinical trial and administered/applied clinical trial drugs/medical devices within 4 weeks before screening
- Those who administered/applied immunosuppressants, chemotherapy, radiation therapy, etc. within 12 weeks before screening
- Persons who have administered cell therapy or gene therapy throughout their lives
- Persons who have administered neurotoxic drugs that can accelerate peripheral nerve damage ① Within 1 week of Screening
- Anti-inflammatory agents or antibiotics: Colchicine, Nitrofurantoin
- Antiretroviral agents: Zalcitabine, Stavudine
- Dichloroacetate ② Within 55 weeks of Screening
- Antiarrhythmic agent: Amiodarone
- Antiparasitic agent: Suramin
- Persons with hypersensitivity to the components of clinical investigational products
- Those who have had metal substances (heart pacemaker, nerve stimulator, cochlear implant, etc.) implanted in their body
- Pregnant, lactating, or planning to become pregnant during the clinical trial period
- people with a psychiatric disorder (anxiety disorder, claustrophobia, or other significant mental disorder) or a history of drug and alcohol abuse that may affect the clinical trial, according to the judgment of the...
- Those who are deemed inappropriate to participate in clinical trials according to the judgment of the investigator
The study team makes the final eligibility decision.
Where it's taking place
- Gyeongju, South Korea
- Seoul, South Korea
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 19 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Gyeongju, South Korea; Seoul, South Korea. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.