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Tests treatment safety and results for Charcot-Marie-Tooth Disease, Type 1A
Official title A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.
ClinicalTrials.gov ID: NCT07140614
What this study is testing
What is EDK060, dose A?
EDK060, dose A is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for charcot-marie-tooth disease, type 1a.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.
- Phase 1: an early, usually small safety study
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 60
You may be able to join 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...
- 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...
You likely can't join 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...
- 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...
- 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...
See the full eligibility 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...
- 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.
- 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Ottawa, Ontario, Canada
- Montreal, Quebec, Canada
- Sherbrooke, Quebec, Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 60 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Ottawa, Ontario, Canada; Montreal, Quebec, Canada; Sherbrooke, Quebec, Canada. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.