NCT07140614

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).

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 28 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.

Conditions

• Charcot-Marie-Tooth Disease, Type 1A

Interventions

Type	Name	Description
DRUG	EDK060, dose A	EDK060, dose A, Single dose, IV infusion
DRUG	EDK060, dose B	EDK060, dose B, Single dose, IV infusion
DRUG	EDK060, dose C	EDK060, dose C, Single dose, IV infusion
DRUG	EDK060, dose D	EDK060, dose D, Single dose, IV infusion
OTHER	Placebo	Placebo

Timeline

• Start date: 2025-09-30
• Primary completion: 2028-02-25
• Completion: 2028-02-26
• First posted: 2025-08-24
• Last updated: 2026-02-27

Locations

3 sites across 1 country: Canada

Regulatory

• FDA-regulated drug study