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
Location: See all (3) locations...
Intervention Type: Other, Drug
Study Type: Interventional
Study Phase: Phase 1
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 60
Healthy Volunteers: f
View:
• 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 motor) in at least one extremity at screening.
Locations
Other Locations
Canada
Montreal Neurological Institute
RECRUITING
Montreal
Novartis Investigative Site
RECRUITING
Ottawa
CIUSS de l´Estrie-CHUS- Hôpital Fleurimont
RECRUITING
Sherbrooke
Contact Information
Primary
Novartis Pharmaceuticals
novartis.email@novartis.com
1-888-669-6682
Backup
Novartis Pharmaceuticals
+41613241111
Time Frame
Start Date: 2025-09-30
Estimated Completion Date: 2028-02-26
Participants
Target number of participants: 28
Treatments
Experimental: EDK060
Doses A; B; C; D
Placebo_comparator: Placebo
Matching placebo
Related Therapeutic Areas
Sponsors
Leads: Novartis Pharmaceuticals