Phase 1 Peripheral Neuropathy Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You are willing and able to sign a consent form before any study assessments begin.
- You are male or female and between 18 and 60 years old (inclusive) at the time of initial screening.
- You have been formally diagnosed with Charcot-Marie-Tooth Disease Type 1A (CMT1A), confirmed by a certified genetic test showing a duplication of the PMP22 gene.
- Nerve signal tests on at least one of your arms show detectable activity (either sensory or movement-related signals).
- Your CMT1A symptom severity score (called CMTNSv2R) is between 3 and 20, confirmed at your baseline visit or recorded in your medical notes within the past 6 months.
- You have measurable muscle weakness in foot/ankle lifting in at least one leg, confirmed at screening or recorded in your medical notes within the past 6 months.
Who may not be able to join:
- You have difficulty communicating with the study team or would be unable to follow the study visits and procedures.
- You have a history of heart, kidney, liver, blood, or immune system conditions.
- You are pregnant or breastfeeding, or are a woman capable of becoming pregnant and not using a highly effective form of contraception throughout the study and follow-up period.
- You are a sexually active male who is unwilling to use a condom during intercourse for the duration of the study.
- You are unwilling or unable to provide a series of small skin samples (skin biopsies) as required by the study.
- You are unwilling or unable to have repeated blood draws, or the study doctor believes these procedures would put you at increased risk.
- You have taken any medication intended to treat or slow CMT1A in the 6 months before screening, including a combination drug called PTX-3003.
- You have had a nerve injury caused by pressure or compression in the past 6 months, or you have another condition that can affect the nerves — such as diabetes, chronic heavy alcohol use, thyroid disease, certain infections, or past exposure to toxic substances or medications that can harm nerves.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Phone: 1-888-669-6682
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Can't join this trial?
Data last synced from ClinicalTrials.gov: 26 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.