A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.

NCT07140614 | Recruiting Phase 1 FDA Drug

• 1 other identifier: CEDK060A12101
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 3

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

Keywords

CMT1A

Outcome Measures

Primary Outcomes (1)

• Incidence 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.

  From baseline up to day 140

Secondary Outcomes (3)

• EDK060 Plasma PK parameters - Cmax

  From baseline up to day 140

• EDK060 Plasma PK parameters - Tmax

  From baseline up to day 140

• EDK060 Plasma PK parameters - AUClast

  From baseline up to day 140

Study Arms (2)

EDK060

EXPERIMENTAL

Doses A; B; C; D

Drug: EDK060, dose A; Drug: EDK060, dose B; Drug: EDK060, dose C; Drug: EDK060, dose D

Placebo

PLACEBO COMPARATOR

Matching placebo

Other: Placebo

Interventions

EDK060, dose C DRUG

EDK060, dose C, Single dose, IV infusion

EDK060

EDK060, dose D DRUG

EDK060, dose D, Single dose, IV infusion

EDK060

Placebo OTHER

Placebo

Placebo

EDK060, dose A DRUG

EDK060, dose A, Single dose, IV infusion

EDK060

EDK060, dose B DRUG

EDK060, dose B, Single dose, IV infusion

EDK060

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (3)

The Ottawa Hospital

Ottawa, Ontario, K1H 8L6, Canada

RECRUITING

Montreal Neurological Institute

Montreal, Quebec, H3A 2B4, Canada

RECRUITING

CIUSS de l´Estrie-CHUS- Hôpital Fleurimont

Sherbrooke, Quebec, J1H 5N4, Canada

RECRUITING

MeSH Terms

Conditions

Charcot-Marie-Tooth Disease

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

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

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 22, 2025
• First Posted: August 24, 2025
• Study Start: September 30, 2025
• Primary Completion (Estimated): February 25, 2028
• Study Completion (Estimated): February 26, 2028
• Last Updated: April 21, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CA (3)