Ignite Creation Date:
2024-05-06 @ 7:59 PM
Last Modification Date:
2024-10-26 @ 3:18 PM
Study NCT ID:
NCT06218134
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-01-23
First Post:
2023-12-19
Brief Title:
Evaluate the Safety and Potential Efficacy of Human Whartons Jelly-derived Mesenchymal Stem Cells With Charcot-Marie-Tooth Disease Type 1E
Sponsor:
Samsung Medical Center
Organization:
Samsung Medical Center
Study Overview
Official Title:
Evaluate the Safety and Potential Efficacy of Human Whartons Jelly-derived Mesenchymal Stem Cells EN001 in Patients With Charcot-Marie-Tooth Disease Type 1E
Status:
NOT_YET_RECRUITING
Status Verified Date:
2023-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None