A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.

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.

Inclusion Criteria:

• 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.

Exclusion Criteria

• Unable to communicate well with the investigator, to understand and comply with the visits and procedures of the study.
• History of cardiac, renal, liver, hematological, immune system disorders.
• Pregnant/nursing female participants. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for the duration of the follow-up period. Sexually active males unless they use a condom during intercourse.
• Inability or unwillingness to provide serial skin biopsy samples.
• Inability or unwillingness to undergo repeated venipuncture or in the opinion of the investigator, participant would be at an increased risk for adverse events related to these procedures.
• Use of any drug intended to modify the course of CMT1A within 6 months from screening, including but not limited to: PTX-3003 (baclofen, sorbitol, and naltrexone in combination).
• History of compression neuropathy within the last 6 months from screening and/or other conditions that can cause peripheral neuropathy, including but not limited to diabetes, chronic alcoholism, exposure to neurotoxic medications, exposure to environmental neurotoxins, traumatic nerve injury, thyroid disease, or infections.

Other protocol-defined inclusion/exclusion criteria may apply