Recruiting
Phase 1

EDK060

Sponsor:

Novartis Pharmaceuticals

Code:

NCT07140614

Conditions

Charcot-Marie-Tooth Disease, Type 1A

Eligibility Criteria

Sex: All

Age: 18 - 60

Healthy Volunteers: Not accepted

Interventions

EDK060, dose A

EDK060, dose B

EDK060, dose C

EDK060, dose D

Placebo

Study Details

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.

Conditions

Charcot-Marie-Tooth Disease, Type 1A

Study ID

NCT07140614

Start date

Sep 30, 2025

Status verified date

Jul, 2026

Completion date

Feb 26, 2028

Anticipated

Primary completion date

Feb 25, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 60

Healthy Volunteers: Not accepted

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/or motor) in at least one extremity at screening.
  • CMTNSv2R score >2 and ≤20 in at least one assessment, confirmed either at the baseline visit or documented in the medical record within the 6 months prior to baseline.
  • Muscle weakness evidenced by foot dorsiflexion MRC grade <5 in at least one limb, confirmed either at screening assessment or documented in the medical record within the 6 months prior to 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

Study Design

Enrollment

28 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: EDK060

Doses A; B; C; D

placebo comparator: Placebo

Matching placebo

Interventions

EDK060, dose A

EDK060, dose A, Single dose, IV infusion

EDK060, dose B

EDK060, dose B, Single dose, IV infusion

EDK060, dose C

EDK060, dose C, Single dose, IV infusion

EDK060, dose D

EDK060, dose D, Single dose, IV infusion

Placebo

Placebo

Primary outcome measure

  • Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: From baseline up to day 140 ]

Central Contacts and Locations

Central contacts

Novartis Pharmaceuticals

1-888-669-6682novartis.email@novartis.com

Novartis Pharmaceuticals

+41613241111

Locations

The Ottawa Hospital

Recruiting

Ottawa, Ontario, Canada, K1H 8L6

Contacts

Jessica Belanger

613-798-5555jessicabelanger@ohri.ca

Principal Investigator:

Alberto Aleman

Novartis Investigative Site

Recruiting

Montreal, Quebec, Canada, H2X 1R9

Montreal Neurological Institute

Recruiting

Montreal, Quebec, Canada, H3A 2B4

Contacts

Julien Kridelka

514 398 78 58Julien.kridelka@mcgill.ca

Principal Investigator:

Rami Massie

CIUSS de l´Estrie-CHUS- Hôpital Fleurimont

Recruiting

Sherbrooke, Quebec, Canada, J1H 5N4

Contacts

Caroline Cayer

1-819-346-1110caroline.cayer.ciussse-chus@ssss.gouv.qc.ca

Principal Investigator:

Marie-France Rioux

More Information

Sponsor

Novartis Pharmaceuticals

Last update posted

Jul 29, 2026

Last verified

Jul, 2026

Keywords

  • CMT1A

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Novartis Pharmaceuticals on 2026-07-29.