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)
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.
Genetic diagnosis: CMT1A type
Those with a CMT Neuropathy Score version 2 (CMTNSv2) between 2 or more and 20 or less.
Those with the following comorbidities confirmed at the time of screening
Subjects with neuromuscular diseases other than CMT1A or neuropathy- inducing factors (uremia) that may affect the safety and efficacy evaluation of this clinical trial, according to the judgment of the investigator.
Individuals diagnosed with type 1 or type 2 diabetes
Individuals diagnosed with active pulmonary tuberculosis
Parallel
EN001
Drug* Cohort 1: EN001 Low dose administered intravenously (IV) 2 times at 4 week intervals. * Cohort 2: EN001 High dose administered intravenously (IV) 2 times at 4 week intervals.
EN001 Placebo
DrugEN001 Placebo administered intravenously (IV) 2 times at 4 week intervals.
[Phase 1b] Dose limiting toxicity (DLT) and adverse drug reactions related to discontinuation of investigational product administration
Present the frequency and percentage of dose-limiting toxicity (DLT) occurrence across dose cohorts, along with detailed information on the types of DLTs. Adverse events related to discontinuation of clinical investigational drug administration, discontinuation of clinical investigational drug administration Regarding related adverse drug reactions, the number of subjects in each cohort, incidence rate (%), and Two-sided 95% confidence intervals and number of occurrences are presented.
[Phase 2a] Change from baseline in the CMT Neuropathy Score (CMTNSv2) at Week 24.
For all efficacy outcome measures, descriptive statistics (number of subjects, mean, standard deviation, median, minimum, and maximum) will be presented by treatment group and assessment time point, along with two-sided 95% confidence intervals.
[Phase1b, Phase2a] CMTNSv2 score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
[Phase1b, Phase2a] CMT examination score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
[Phase1b, Phase2a] Rasch-modified CMTNSv2 score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
[Phase1b, Phase2a] Rasch-modified CMTES score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
