CEDK060A12101: FIH study of EDK060 in CMT1a
Research type
Research Study
Full title
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).
IRAS ID
1013420
Contact name
Sakshi Bhardwaj
Contact email
Sponsor organisation
Novartis Pharma UK Ltd.
Clinicaltrials.gov Identifier
Research summary
This study is designed to evaluate a potential new treatment called EDK060 for Charcot Marie Tooth Type 1A (CMT1A). Its main goals are to evaluate whether EDK060 is well tolerated, and capable of lowering PMP22 protein levels when administered intravenously. This is the first time EDK060 will be tested in humans. Approximately 28 adults aged 18 to 60 with CMT1A across two countries will be included. The study follows a controlled design in which a single dose of either EDK060 or a placebo is given. The total study duration is about 5–6 months and includes around 12 scheduled visits for clinical evaluations. At these visits, physical and neurological examinations are performed, heart function is monitored by ECG, and blood and urine samples are collected for safety assessments and research analyses. Questionnaires on health and disease impact are completed, and additional procedures such as nerve conduction tests, movement related assessments, and small skin sample collections are carried out at some of the visits. These activities are intended to help researchers understand how the treatment behaves in the body, how well it reaches target tissues, and how CMT1A progresses over time. As EDK060 is being tested in humans for the first time, the full range of potential side effects is not yet known, and risks may also arise from the study procedures themselves. While no direct medical benefit is expected, the information gained may support future therapeutic development for CMT1A. Eligibility for participation depends on meeting specific health and study related criteria identified during screening assessments.
REC name
London - Harrow Research Ethics Committee
REC reference
26/LO/0081
Date of REC Opinion
14 Apr 2026
REC opinion
Further Information Favourable Opinion