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Recruiting NCT07140614

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

Phase I Interventional Charcot-Marie-Tooth Disease, Type 1A

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: EDK060, dose A, EDK060, dose B, EDK060, dose C, EDK060, dose D.
Who it may be relevant to
Registry conditions: Charcot-Marie-Tooth Disease, Type 1A. Basic parameters: 18 years — 60 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Canada
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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).

Overview

The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.

Interventions

  • Drug EDK060, dose A
    EDK060, dose A, Single dose, IV infusion
  • Drug EDK060, dose B
    EDK060, dose B, Single dose, IV infusion
  • Drug EDK060, dose C
    EDK060, dose C, Single dose, IV infusion
  • Drug EDK060, dose D
    EDK060, dose D, Single dose, IV infusion
  • Other Placebo
    Placebo

Primary outcome measures

  • Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time frame: From baseline up to day 140]
Secondary outcome measures (3)
  • EDK060 Plasma PK parameters - Cmax [Time frame: From baseline up to day 140]
  • EDK060 Plasma PK parameters - Tmax [Time frame: From baseline up to day 140]
  • EDK060 Plasma PK parameters - AUClast [Time frame: From baseline up to day 140]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Triple blind
Primary purpose
Treatment

Study locations

Canada · 4 centers
  • The Ottawa Hospital — Ottawa
  • Novartis Investigative Site — Montreal
  • Montreal Neurological Institute — Montreal
  • CIUSS de l´Estrie-CHUS- Hôpital Fleurimont — Sherbrooke

Identifiers

NCT: NCT07140614 · CEDK060A12101

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗