A First-in-human, Multi-center, Randomized, Participant- and Investigator-blinded, Placebo Controlled, Single Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With Charcot-Marie Tooth Type 1A (CMT1A).
The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.
• Provide written informed consent before any assessment is performed.
• Be male or female and 18 to 60 (inclusive) years of age at the time of screening.
• Participant must have a clinical diagnosis of Charcot-Marie-Tooth Disease Type 1A (CMT1A) including verified documentation of genetic testing showing duplication of the PMP22 gene by an accredited/certified laboratory (according to local regulations)
• Have detectable upper extremity nerve conduction velocities (sensory and/or motor) in at least one extremity at screening.
• CMTNSv2R score \>2 and ≤20 in at least one assessment, confirmed either at the baseline visit or documented in the medical record within the 6 months prior to baseline.
• Muscle weakness evidenced by foot dorsiflexion MRC grade \<5 in at least one limb, confirmed either at screening assessment or documented in the medical record within the 6 months prior to screening.