First human testing of EDK060 in adults with CMT1A.

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a first-in-human Phase 1 interventional study in which adults with genetically confirmed CMT1A will receive one of four doses of EDK060 or placebo. Participants will be monitored closely for safety, tolerability, and pharmacokinetics after dosing and followed for adverse events and selected neurological measures. Key eligibility includes age 18–60, PMP22 gene duplication, measurable nerve conduction, a CMTNSv2R score >2 and ≤20, and foot dorsiflexion weakness. The trial is sponsored by Novartis and conducted at sites in Ottawa, Montreal, and Sherbrooke.

Why it matters

Who can join: the full rules

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/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.

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.

Other protocol-defined inclusion/exclusion criteria may apply

Where it's running

• The Ottawa Hospital
  Ottawa, Ontario, Canada Recruiting
• Novartis Investigative Site
  Montreal, Quebec, Canada Recruiting
• Montreal Neurological Institute
  Montreal, Quebec, Canada Recruiting
• CIUSS de l´Estrie-CHUS- Hôpital Fleurimont
  Sherbrooke, Quebec, Canada Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.