SMC
Seoul, Gangnam-gu, 06351, South Korea
NCT Number: NCT06218134
Charcot-Marie-Tooth disease (CMT) is a hereditary peripheral nerve disease that causes causes muscle atrophy, muscle weakness, sensory loss, balance disorder, gait disorder, blindness, hearing disorder, breathing disorder, vocal cord paralysis, foot deformity, scoliosis, and reflex dysfunction, More than 140 types of genes causing this disease are known. Charcot-Marie-Tooth (CMT) 1E, the target disease of this study, shows very severe symptoms compared to other Charcot-Marie-Tooth types. In cases of early onset, especially in children under 5 years of age, almost all patients are unable to walk without a wheelchair and have severe illness. Symptoms include scoliosis, breathing problems, vocal cord paralysis, foot deformity, loss of sensation and reflex function.
Additionally, more than 40% of Charcot-Marie-Tooth (CMT) 1E patients have hearing loss and become unable to live without hearing aids. Although this disease is very disabling, there is still no approved treatment.
To date, there is a lack of practical treatment or treatment support methods that can change the progression of hereditary motor and sensory neuropathy, so the focus is on pain control, use of assistive devices, and rehabilitation treatment, but the treatment effect is almost non-existent.
This study is conducted for the purpose of confirming the safety and exploratory treatment effect by administering EN001, an allogeneic umbilical cord-derived mesenchymal stem cell, once intravenously to patients with Charcot-Marie-Tooth (CMT) 1E.
EN001 is an allogeneic (alien-derived) umbilical cord-derived mesenchymal stem cell, and a phase 1 clinical trial of single intravenous administration was completed in 9 Charcot-Marie-Tooth (CMT) type 1A patients. Among the four adverse reactions that occurred in the participating research subjects, there were no adverse drug reactions related to EN001, and all four cases were mild and recovered. No serious adverse drug reactions or infusion reactions were observed in any study subjects, so this is a safe stem cell treatment.
Through efficacy tests and non-clinical tests, the effectiveness of improving behavior and increasing nerve and motor conduction speeds when administering the test drug to animal models of muscle disease was confirmed, so it is expected that this study can stabilize the disease progression in patients, and it will contribute to improving the quality of life and further promoting public health and welfare.
Looking for future studies?
Notify Me19 year and older
All sexes
Interventional
Not applicable
Seoul, Gangnam-gu, 06351, South Korea
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
Exclusion criteria
⑤ Serum virus test (HBsAg, anti-HBc, anti-HCV, Positive for any one of HIV Ag/Ab)
<!-- -->
<!-- -->
human Wharton's jelly-derived mesenchymal stem cells(product name: EN001)
Time frame: up to 12month
Check Fever, high blood pressure, low blood pressure, tachycardia, hyperventilation, rash, shock-related reactions related to administration
Time frame: up to 12month
Charcot-Marie-Tooth disease 1E disease scale: CMTNS(Charcot-Marie-Tooth Neuropathy Score version 2), Lower values indicate better clinical conditions.
Samsung Medical Center
Other
Evaluate the Safety and Potential Efficacy of Human Wharton's Jelly-derived Mesenchymal Stem Cells (EN001) in Patients With Charcot-Marie-Tooth Disease Type 1E
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
Published trials that share one or more normalized conditions with this study.
NCT07335822
Charcot-Marie-Tooth Disease, Congenital Abnormalities
Istanbul, Fatih, Turkey (Türkiye)
View Trial DetailsNCT01918826
Charcot Marie Tooth Disease, Charcot-Marie-Tooth Disease
Nice, France
View Trial DetailsNCT03386266
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth Disease, Type IA
Goettigen, Lower Saxony, Germany
View Trial DetailsNCT07408596
Carpal Tunnel Syndrome, Carpal Tunnel Syndrome (CTS)
Istanbul, Fatih, Turkey (Türkiye)
View Trial Details