Testing EDK060, dose A with EDK060, dose B against a placebo for type 1A charcot-marie-tooth disease
Official title: A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.
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).
- Phase 1
- 2 groups
- Sites in Ottawa, Montreal and 1 more city
- Recruiting
Interventions (5)
- Medication
EDK060, dose A
EDK060, dose A, Single dose, IV infusion
- Medication
EDK060, dose B
EDK060, dose B, Single dose, IV infusion
- Medication
EDK060, dose C
EDK060, dose C, Single dose, IV infusion
- Medication
EDK060, dose D
EDK060, dose D, Single dose, IV infusion
- Other intervention
Placebo
Placebo
Canadian Sites (4)
4 of 4 recruiting
- Recruiting
The Ottawa Hospital
Ottawa, Ontario
- Recruiting
Novartis Investigative Site
Montreal, Quebec
- Recruiting
Montreal Neurological Institute
Montreal, Quebec
- Recruiting
CIUSS de l´Estrie-CHUS- Hôpital Fleurimont
Sherbrooke, Quebec
Eligibility Criteria
See who this study is looking for23 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Be male or female and 18 to 60 (inclusive) years of age at the time of screeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
- +Provide written informed consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more → before any assessment is performed.
- +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 visitBaselineYour starting measurements, taken before treatment begins.Read more → 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 CriteriaExclusion criteriaThe things that would prevent someone from taking part.Read more →
- +Unable to communicate well with the investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →, to understand and comply with the visits and procedures of the study.
- +History of cardiac, renal, liver, hematological, immune system disorders.
- +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 eventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → 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 protocolProtocolThe detailed plan a study must follow.Read more →-defined inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more →/exclusion criteria may apply
- +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 periodFollow-upContinued check-ins after the treatment part is finished.Read more →. Sexually active males unless they use a condom during intercourse.
Exclusion
- −Unable to communicate well with the investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →, to understand and comply with the visits and procedures of the study.
- −History of cardiac, renal, liver, hematological, immune system disorders.
- −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 eventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → related to these procedures.
- −Use of any drug intended to modify the course of CMT1A within 6 months from screeningScreeningThe checks done before joining, to see whether a study fits.Read more →, 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 protocolProtocolThe detailed plan a study must follow.Read more →-defined inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more →/exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → may apply
- −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 periodFollow-upContinued check-ins after the treatment part is finished.Read more →. Sexually active males unless they use a condom during intercourse.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 1Phase 1The earliest stage of human testing, in a small group, focused on safety.Read more → — an early safety study in a small group, checking how it is tolerated and at what dose.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 2 groups in this study.
Group A receives one or more of: EDK060, dose A, EDK060, dose B, EDK060, dose C and EDK060, dose D.
Registry label: A: EDK060
Group B, the placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group, receives Placebo.
Registry label: B: Placebo
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
You, the study team, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
One group receives a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → — a dummy treatment with no active medicine in it.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 18 years to 60 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 28 people.
The study is currently expected to finish around February 2028.
The main measurement is taken over: From baselineBaselineYour starting measurements, taken before treatment begins.Read more → up to day 140.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Incidence and severity of Adverse EventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → (AEs) and Serious Adverse EventsSerious adverse eventAn adverse event meeting a formal severity threshold, such as requiring hospital admission.Read more → (SAEs) — measured over From baselineBaselineYour starting measurements, taken before treatment begins.Read more → up to day 140.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT07140614 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About EDK060, dose ADrug
EDK060, dose A, Single dose, IV infusion
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About EDK060, dose BDrug
EDK060, dose B, Single dose, IV infusion
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About EDK060, dose CDrug
EDK060, dose C, Single dose, IV infusion
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About EDK060, dose DDrug
EDK060, dose D, Single dose, IV infusion
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About PlaceboOther
Placebo
From the trial registry — its own words, unedited.
What a other is here: An intervention the registry did not place in another category.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 18 years to 60 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
Yes. This study includes a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group — a dummy treatment with no active medicine in it.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
You, the study team, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 18 years to 60 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: From baselineBaselineYour starting measurements, taken before treatment begins.Read more → up to day 140.
The study as a whole is currently expected to finish around 2028-02-26.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 28 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 3 locations, including: Ottawa, Ontario, Canada; Montreal, Quebec, Canada; Sherbrooke, Quebec, Canada.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.