Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A : An Open, Dose-escalation, Phase 1b Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A (CMT1A)
This clinical trial is a phase 1b clinical trial with a 3+3 dose escalation design to evaluate the safety, including tolerability, of EN001 and explore efficacy.The study was designed using the traditional 3+3 dose escalation method to confirm the maximum tolerated dose (MTD) and determine the recommended phase 2 dose (RP2D).Dose increase is carried out until the maximum tolerated dose (MTD) is confirmed at a dose of 2.5 × 10^6 cells/kg (Cohort 2) or less, which is the maximum planned dose (MPD). The maximum tolerated dose (MTD) is defined as the highest dose at which the incidence of dose limiting toxicity (DLT) is lower than 33%. To determine the maximum tolerated dose (MTD), 3-6 test subjects from each dose cohort are enrolled and EN001 is administered twice at 4-week intervals, and dose-limiting toxicity (DLT) is evaluated until 4 weeks (visit 6).The safety review committee (SRC) is comprised of the principal investigator, sponsor, etc. as members, and EN001 confirmed by the end of each cohort (end of dose-limiting toxicity (DLT) evaluation of the last dosed subject in the cohort). Safety data are comprehensively reviewed to determine all matters related to dose, such as increase or decrease in dose, and finally the recommended phase 2 dose (RP2D) is determined.In addition, test subjects participating in phase 1b will be followed up for safety and effectiveness for 5 years from the time of EN001 administration according to the long-term follow-up protocol..
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Klinische Studie| Erscheinungsjahr: |
2024 |
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| Erschienen: |
2024 |
| Enthalten in: |
ClinicalTrials.gov - (2024) vom: 25. März Zur Gesamtaufnahme - year:2024 |
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| Sprache: |
Englisch |
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| Links: |
Volltext [kostenfrei] |
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| Themen: |
610 |
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| Anmerkungen: |
Source: Link to the current ClinicalTrials.gov record., First posted: March 25, 2024, Last downloaded: ClinicalTrials.gov processed this data on April 03, 2024, Last updated: April 03, 2024 |
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| Study ID: |
NCT06328712 |
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| Veröffentlichungen zur Studie: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
CTG00015797X |
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| 245 | 1 | 0 | |a Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A |b An Open, Dose-escalation, Phase 1b Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A (CMT1A) |
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| 520 | |a This clinical trial is a phase 1b clinical trial with a 3+3 dose escalation design to evaluate the safety, including tolerability, of EN001 and explore efficacy.The study was designed using the traditional 3+3 dose escalation method to confirm the maximum tolerated dose (MTD) and determine the recommended phase 2 dose (RP2D).Dose increase is carried out until the maximum tolerated dose (MTD) is confirmed at a dose of 2.5 × 10^6 cells/kg (Cohort 2) or less, which is the maximum planned dose (MPD). The maximum tolerated dose (MTD) is defined as the highest dose at which the incidence of dose limiting toxicity (DLT) is lower than 33%. To determine the maximum tolerated dose (MTD), 3-6 test subjects from each dose cohort are enrolled and EN001 is administered twice at 4-week intervals, and dose-limiting toxicity (DLT) is evaluated until 4 weeks (visit 6).The safety review committee (SRC) is comprised of the principal investigator, sponsor, etc. as members, and EN001 confirmed by the end of each cohort (end of dose-limiting toxicity (DLT) evaluation of the last dosed subject in the cohort). Safety data are comprehensively reviewed to determine all matters related to dose, such as increase or decrease in dose, and finally the recommended phase 2 dose (RP2D) is determined.In addition, test subjects participating in phase 1b will be followed up for safety and effectiveness for 5 years from the time of EN001 administration according to the long-term follow-up protocol. | ||
| 650 | 2 | |a Tooth Diseases | |
| 650 | 2 | |a Charcot-Marie-Tooth Disease | |
| 650 | 2 | |a Nerve Compression Syndromes | |
| 650 | 2 | |a Hereditary Sensory and Motor Neuropathy | |
| 650 | 2 | |a Disease | |
| 650 | 4 | |a Study Type: Interventional | |
| 650 | 4 | |a Recruitment Status: Recruiting | |
| 650 | 4 | |a Phase: Phase 1 | |
| 650 | 4 | |a 610 | |
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