A Study to Assess the Efficacy and Safety of PXT3003 in Charcot-Marie-Tooth Type 1A (NCT05092841) | Clinical Trial Compass
CompletedPhase 3
A Study to Assess the Efficacy and Safety of PXT3003 in Charcot-Marie-Tooth Type 1A
China176 participantsStarted 2021-09-28
Plain-language summary
This is a randomized, double-blind, placebo-controlled and multicenter 3 phase trial evaluating the therapeutic effect and safety of CMT1A by PXT3003. This double-blind study will assess in parallel groups 1 dose of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.
Who can participate
Age range16 Years – 65 Years
SexALL
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Inclusion criteria
✓. Patients aged 16 to 65 years (included boundary value), of either sex;
✓. Patients with CMT1A (PMP22 duplication on chromosome 17p11.2) confirmed by gene diagnosis; 3.2 \< CMTNS-v2 score ≤ 18;
✓.Patients are dorsalis pedis flexor weakness at least (clinical evaluation); 5.Ulnar nerve motor nerve conductance velocity \> 15 m/s; 6.Subjects participate in clinical trials and sign informed consent voluntarily , and they have the ability to understand as will as abide by research procedures.
Exclusion criteria
✕. Being allergic to RS-baclofen, naltrexone HCL, D-sorbitol or any component in pxt3003 excipients or having other serious prior allergic reaction;
✕. Existence contraindications of baclofen, naltrexone or sorbitol, such as porphyria;
✕. Any other associated cause of peripheral neuropathy such as diabetes mellitus (including diabetes history and glycosylated hemoglobin \>6.5%)
✕. Subjects with other neurological diseases affecting the evaluation of study treatment;
✕. Patients with the score of ONLS score is 0;
✕. A history of unstable medical diseases with clinically significant unstable medical diseases (unstable angina pectoris, tumor, blood disease, hepatitis or liver failure, renal failure, etc.) that may cause harm to the subjects participating in this study in the past 1 year;
✕. Limb surgery had implemented within the first six months of randomization or will be planned before the completion of the clinical trial;