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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
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dose group A (Low dose) | Experimental | Participants will receive EN001 intravenously (IV) once on Day 0. |
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| Dose group B (High dose) | Experimental | Participants will receive EN001 intravenously (IV) once on Day 0. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| EN001 | Drug | EN001 intravenously (IV) in the treatment of Charcot-Marie-Tooth disease (CMT) type 1A Dosage for each group is as follows. Dose group A (Low dose): 5.0x10^5 cells/kg |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants of any Adverse Events (AEs)/Serious Adverse Events (SAEs) related investigational product | Number of participants with treatment-related AEs/SAEs as assessed by CTCAE v5.0 | Week 16 after treatment |
| Determination of Dose-limiting toxicity (DLT) levels of EN001 | Among the adverse events occurring for 4 weeks after administration of the investigational product, Grade 3 or higher adverse events according to CTCAE v5.0 | Up to Week 4 after dosing on Day 0 |
| Determination of Maximum tolerated dose (MTD) levels of EN001 | Among the adverse events occurring for 4 weeks after administration of the investigational product, Grade 3 or higher adverse events according to CTCAE v5.0 Maximum tolerated dose defines the evaluated maximum dose level in which greater than two participants of six participants experience Dose-limiting toxicity (DLT) under the dose level. The dose level where two participants of six participants experience DLT will be the maximum tolerated dose. | Up to Week 4 after dosing on Day 0 |
| Number of participants with Vital Signs abnormalities | Vital Signs include blood pressure (mmHg), pulse (times/minute), respiratory rate (times/minute), and body temperature (℃) and will be assessed by CTCAE v 5.0 to evaluate safety and tolerability of EN001. The number of participants with at least one potentially clinically significant abnormal vital sign finding were reported as treatment emergent adverse events (TEAEs). | From screening up to Week 16 |
| Number of participants with clinically significant abnormalities of Physical Examinations | Physical Examinations include general appearance, head, ears/eyes/nose/throat, cardiovascular, respiratory, abdomen, skin, lymph nodes, extremities, musculoskeletal and neurologic and will be assessed by CTCAE v 5.0 to evaluate safety and tolerability of EN001. Number of participants with potentially clinically significant abnormalities in physical examinations were reported as TEAEs. |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of adverse events (AEs) | Occurrence of any adverse reactions, development of new blood clots, tumors, immune responses (like autoimmune reactions) and death, and/or serious adverse events related investigational product will be summarized by actual treatment groups respectively. | From screening to the end of treatment/withdrawal visit (up to approximately 5 years per subject) |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Samsung Medical Center | Seoul | South Korea |
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| ID | Term |
|---|---|
| D002607 | Charcot-Marie-Tooth Disease |
| ID | Term |
|---|---|
| D015417 | Hereditary Sensory and Motor Neuropathy |
| D009421 | Nervous System Malformations |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
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| EN001 | Drug | EN001 intravenously (IV) in the treatment of Charcot-Marie-Tooth disease (CMT) type 1A Dosage for each group is as follows. Dose group B (High dose): 2.5x10^6 cells/kg |
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| From screening up to Week 16 |
| Number of participants with abnormalities of Laboratory Parameters | Laboratory Parameters include hematology, chemistry laboratory tests, urinalysis, coagulation test and plasma viral load test and will be assessed by CTCAE 5.0 to evaluate safety and tolerability of EN001. Number of participants with at least one potentially clinically significant abnormal finding were reported as TEAEs. | From screening up to Week 16 |
| Number of participants with 12-lead Electrocardiography (ECG) abnormalities | Measured by result of the ECG measurements and findings | From screening to baseline on Day 1 (Predose to end of infusion and 90 min after completion of infusion) |
| Number of participants with abnormalities of Vital Signs, Physical Findings, and Laboratory Parameters | Abnormalities of Vital Signs, Physical Findings, and Laboratory parameters (as described above) will be collected and analyzed, and then assessed by CTCAE 5.0 to evaluate the long-term safety of EN001. | From screening to the end of treatment/withdrawal visit (up to approximately 5 years per subject) |
| Change from baseline in disease severity CMTNS-v2 score | Disease severity measured by CMTNS-v2 score will be collected and analyzed to evaluate the exploratory efficacy of EN001. | Screening and baseline on Day 0 (up to approximately 5 years per subject after Week 4) |
| Change from baseline in gait and balance functions | Gait and balance functions measured by Ten Meter Walking Test(10MWT), Functional Disability Scale (FDS), and Overall Neuropathy Limitation Score (ONLS) leg scale will be collected and analyzed to evaluate the exploratory efficacy of EN001. | Screening and baseline on Day 0 (up to approximately 5 years per subject after Week 4) |
| Change from baseline in the degree of muscle damage (%) | The degree of muscle damage measured by lower extremity magnetic resonance imaging (MRI) scan will be collected and analyzed to evaluate the exploratory efficacy of EN001. | Screening and baseline on Day 0 (up to approximately 5 years per subject after Week 4) |
| Change from baseline in nerve regeneration potential | Nerve generation potential measured by MNCV, SNCV, CMAP, and SNAP will be collected and analyzed to evaluate the exploratory efficacy of EN001. | Screening and baseline on Day 0 (up to approximately 5 years per subject after Week 4) |
| D019636 | Neurodegenerative Diseases |
| D011115 | Polyneuropathies |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |