Status:
RECRUITING
A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Charcot-Marie-Tooth Disease, Type 1A
Eligibility:
All Genders
18-60 years
Phase:
PHASE1
Brief 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 Criteria
Inclusion
- Inclusion Criteria:
- 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.
- Exclusion Criteria
- Unable to communicate well with the investigator, to understand and comply with the visits and procedures of the study.
- History of cardiac, renal, liver, hematological, immune system disorders.
- Pregnant/nursing female participants. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for the duration of the follow-up period. Sexually active males unless they use a condom during intercourse.
- Inability or unwillingness to provide serial skin biopsy samples.
- Inability or unwillingness to undergo repeated venipuncture or in the opinion of the investigator, participant would be at an increased risk for adverse events related to these procedures.
- Use of any drug intended to modify the course of CMT1A within 6 months from screening, including but not limited to: PTX-3003 (baclofen, sorbitol, and naltrexone in combination).
- History of compression neuropathy within the last 6 months from screening and/or other conditions that can cause peripheral neuropathy, including but not limited to diabetes, chronic alcoholism, exposure to neurotoxic medications, exposure to environmental neurotoxins, traumatic nerve injury, thyroid disease, or infections.
- Other protocol-defined inclusion/exclusion criteria may apply
Exclusion
Key Trial Info
Start Date :
September 30 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
February 26 2028
Estimated Enrollment :
28 Patients enrolled
Trial Details
Trial ID
NCT07140614
Start Date
September 30 2025
End Date
February 26 2028
Last Update
December 11 2025
Active Locations (3)
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1
Novartis Investigative Site
Ottawa, Ontario, Canada, K1H 8L6
2
Montreal Neurological Institute
Montreal, Quebec, Canada, H3A 2B4
3
CIUSS de l´Estrie-CHUS- Hôpital Fleurimont
Sherbrooke, Quebec, Canada, J1H 5N4