A Study to Assess the Efficacy and Safety of PXT3003 in Charcot-Marie-Tooth Type 1A

Ki vehet részt

• 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; 4.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

Ki nem vehet részt

• Being allergic to RS-baclofen, naltrexone HCL, D-sorbitol or any component in pxt3003 excipients or having other serious prior allergic reaction
• Limb surgery had implemented within the first six months of randomization or will be planned before the completion of the clinical trial
• Subjects with concomitant treatment 4 weeks before enrollment, including but not limited to baclofen, naltrexone, sorbitol, opioids, vitamin C, levothyroxine and potentially neurotoxic drugs (such as amiodarone and chloroquine)
• Participated in another clinical trial and used the test drug within the last 30 days
• Subjects with other neurological diseases affecting the evaluation of study treatment
• 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
• Syphilis antibody and HIV antibody positive subjects
• Any other associated cause of peripheral neuropathy such as diabetes mellitus (including diabetes history and glycosylated hemoglobin >6.5%)
• Hepatic or renal dysfunction:; TBIL>1.5×ULN,ALT>3×ULN,AST>3×ULN; Cr>1.5×ULN
• Existence contraindications of baclofen, naltrexone or sorbitol, such as porphyria
• Patients with the score of ONLS score is 0
• Subjects with tumors indicated by chest radiograph or B-ultrasound
• Subjects with alcohol dependence in recent 3 months
• Females that are of childbearing potential, pregnant, or are breast-feeding;Subjects who are unable to use appropriate contraceptives during the trial
• Subjects unable to complete the follow-up of study
• Different subjects from the same family and living in the same residence can only include one subject, so as to avoid treatment confusion, affect blind treatment and affect the interpretation of the research results
• Investigators affirm the compliance in a certain subject is poor or there are some other factors that are not suitable to participate in this clinical trial