The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
28
EDK060, dose A, Single dose, IV infusion
EDK060, dose B, Single dose, IV infusion
EDK060, dose C, Single dose, IV infusion
Novartis Investigative Site
Ottawa, Ontario, Canada
RECRUITINGMontreal Neurological Institute
Montreal, Quebec, Canada
RECRUITINGCIUSS de l´Estrie-CHUS- Hôpital Fleurimont
Sherbrooke, Quebec, Canada
RECRUITINGIncidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Incidence of AEs and SAEs by treatment group, including changes in vital signs, laboratory measures, neurological examinations and electrophysiology measures qualifying and reported as AEs.
Time frame: From baseline up to day 140
EDK060 Plasma PK parameters - Cmax
The maximum (peak) observed EDK060 plasma concentration after single dose administration (mass x volume-1)
Time frame: From baseline up to day 140
EDK060 Plasma PK parameters - Tmax
The time to reach maximum (peak) plasma, EDK060 concentration after single dose administration (time)
Time frame: From baseline up to day 140
EDK060 Plasma PK parameters - AUClast
The area under the concentration-time curve of EDK060 from time zero to the last measurable concentration sampling time (tlast) (mass x time x volume-1)
Time frame: From baseline up to day 140
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EDK060, dose D, Single dose, IV infusion
Placebo