A Phase 1 interventional study of EDK060, dose A and EDK060, dose B in Charcot-Marie-Tooth Disease, Type 1A, sponsored by Novartis Pharmaceuticals. Recruiting at 4 sites in Canada. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2026-07-29.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 1, Interventional, and Treatment
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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.
113 studies on the registry are indexed under Charcot-Marie-Tooth Disease; 37 are open to participants now.
This study's planned enrollment of 28 is below the median of 41 across 62 interventional studies indexed under Charcot-Marie-Tooth Disease.
Browse Charcot-Marie-Tooth Disease studies →Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria
Other protocol-defined inclusion/exclusion criteria may apply
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Doses A; B; C; D
Drug: EDK060, dose A · Drug: EDK060, dose B · Drug: EDK060, dose C · Drug: EDK060, dose D
Matching placebo
Other: Placebo
EDK060, dose A, Single dose, IV infusion
EDK060, dose B, Single dose, IV infusion
EDK060, dose C, Single dose, IV infusion
EDK060, dose D, Single dose, IV infusion
Placebo
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.
Time frame: From baseline up to day 140
EDK060 Plasma PK parameters - Cmax
The maximum (peak) observed EDK060 plasma concentration after single dose administration (mass x volume-1)
Time frame: From baseline up to day 140
EDK060 Plasma PK parameters - Tmax
The time to reach maximum (peak) plasma, EDK060 concentration after single dose administration (time)
Time frame: From baseline up to day 140
EDK060 Plasma PK parameters - AUClast
The area under the concentration-time curve of EDK060 from time zero to the last measurable concentration sampling time (tlast) (mass x time x volume-1)
Time frame: From baseline up to day 140
Plan to share: No
No publications or documents are linked to this record.
Started Sep 2025 (actual). Primary completion is estimated for Feb 2028, 28 months after the start.
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