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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

28 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-09-30

Study Completion Date

2028-02-26

Brief Summary

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

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Detailed Description

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Conditions

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Charcot-Marie-Tooth Disease, Type 1A

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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EDK060

Doses A; B; C; D

Group Type EXPERIMENTAL

EDK060, dose A

Intervention Type DRUG

EDK060, dose A, Single dose, IV infusion

EDK060, dose B

Intervention Type DRUG

EDK060, dose B, Single dose, IV infusion

EDK060, dose C

Intervention Type DRUG

EDK060, dose C, Single dose, IV infusion

EDK060, dose D

Intervention Type DRUG

EDK060, dose D, Single dose, IV infusion

Placebo

Matching placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Placebo

Interventions

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EDK060, dose A

EDK060, dose A, Single dose, IV infusion

Intervention Type DRUG

EDK060, dose B

EDK060, dose B, Single dose, IV infusion

Intervention Type DRUG

EDK060, dose C

EDK060, dose C, Single dose, IV infusion

Intervention Type DRUG

EDK060, dose D

EDK060, dose D, Single dose, IV infusion

Intervention Type DRUG

Placebo

Intervention Type OTHER

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No