Actively Recruiting
A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.
Led by Novartis Pharmaceuticals · Updated on 2026-04-21
28
Participants Needed
3
Research Sites
125 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Provide written informed consent before any assessments
- Be male or female aged 18 to 60 years inclusive at screening
- Have a clinical diagnosis of Charcot-Marie-Tooth Disease Type 1A confirmed by genetic testing showing duplication of the PMP22 gene
- Have detectable upper extremity nerve conduction velocities in at least one extremity at screening
- CMTNSv2R score greater than 2 and less than or equal to 20 in at least one assessment confirmed at baseline or within 6 months prior
- Have muscle weakness shown by foot dorsiflexion MRC grade less than 5 in at least one limb confirmed at screening or within 6 months prior
You will not qualify if you...
- Unable to communicate well or comply with study visits and procedures
- History of cardiac, renal, liver, hematological, or immune system disorders
- Pregnant or nursing women; women of child-bearing potential not using effective contraception; sexually active males not using condoms during intercourse
- Unable or unwilling to provide serial skin biopsy samples
- Unable or unwilling to undergo repeated venipuncture or at increased risk from these procedures
- Use of any drug modifying CMT1A course within 6 months before screening, including PTX-3003
- History of compression neuropathy within 6 months prior or other conditions causing peripheral neuropathy such as diabetes, chronic alcoholism, neurotoxic medication exposure, environmental neurotoxins, traumatic nerve injury, thyroid disease, or infections
- Other protocol-defined inclusion/exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
The Ottawa Hospital
Ottawa, Ontario, Canada, K1H 8L6
Actively Recruiting
2
Montreal Neurological Institute
Montreal, Quebec, Canada, H3A 2B4
Actively Recruiting
3
CIUSS de l´Estrie-CHUS- Hôpital Fleurimont
Sherbrooke, Quebec, Canada, J1H 5N4
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
CONTACT
N
Novartis Pharmaceuticals
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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