Charcot-Marie-Tooth Disease, Type 1APhase 1NCT07140614
A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.
A Phase 1 study testing EDK060, dose A, EDK060, dose B and EDK060, dose C in people with Charcot-Marie-Tooth Disease, Type 1A, run by Novartis Pharmaceuticals at 4 sites in Canada.
Trial summary
The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Charcot-Marie-Tooth Disease, Type 1A
- What is being tested?
- EDK060, dose A, EDK060, dose B and EDK060, dose C
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 28 participants
- Where
- 4 sitesCanada
- Study dates
- 30 Sep 2025to 26 Feb 2028
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
Canada4 sites, 4 recruiting
- Recruiting
Novartis Investigative Site
Montreal, Quebec, H2X 1R9
- Recruiting
Montreal Neurological Institute
Montreal, Quebec, H3A 2B4
Julien Kridelka, 514 398 78 58, Julien.kridelka@mcgill.ca
Rami Massie
- Recruiting
The Ottawa Hospital
Ottawa, Ontario, K1H 8L6
Jessica Belanger, 613-798-5555 ext. 19627, jessicabelanger@ohri.ca
Alberto Aleman
- Recruiting
CIUSS de l´Estrie-CHUS- Hôpital Fleurimont
Sherbrooke, Quebec, J1H 5N4
Caroline Cayer, 1-819-346-1110 ext. 13920, caroline.cayer.ciussse-chus@ssss.gouv.qc.ca
Marie-France Rioux
About this study
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).
The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.
- Conditions
- Charcot-Marie-Tooth Disease, Type 1A
- Being tested
- EDK060, dose A, EDK060, dose B, EDK060, dose C, EDK060, dose D, Placebo
- Keywords
- CMT1A
- Medical subject headings
- Charcot-Marie-Tooth Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EDK060, dose ADrug
EDK060, dose A, Single dose, IV infusion
EDK060, dose BDrug
EDK060, dose B, Single dose, IV infusion
EDK060, dose CDrug
EDK060, dose C, Single dose, IV infusion
EDK060, dose DDrug
EDK060, dose D, Single dose, IV infusion
PlaceboOther
Placebo
| Study group | Role | What they receive |
|---|---|---|
| EDK060 | Experimental | Doses A; B; C; D Receives: Drug: EDK060, dose A; Drug: EDK060, dose B; Drug: EDK060, dose C; Drug: EDK060, dose D |
| Placebo | Placebo comparator | Matching placebo Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From baseline up to day 140
Secondary outcomes (3)
EDK060 Plasma PK parameters - Cmax
The maximum (peak) observed EDK060 plasma concentration after single dose administration (mass x volume-1)
Measured: 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)
Measured: 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)
Measured: From baseline up to day 140
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2025
- Study start30 Sep 2025
- Primary completion25 Feb 2028 (estimated)
- Study completion26 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Charcot-Marie-Tooth Disease, Type 1AStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 28 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07140614
- Sponsor's protocol ID
- CEDK060A12101
- Status last verified
- Jul 2026
- First submitted
- 22 Aug 2025
- Last updated
- 29 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No