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

Last updated: January 7, 2025
Sponsor: Tasly GeneNet Pharmaceuticals Co., Ltd
Overall Status: Completed

Phase

3

Condition

Peripheral Neuropathy

Neuropathy

Treatment

PXT3003 placebo

PXT3003

Clinical Study ID

NCT05092841
TSL-CM-PXT3003-Ⅲ
  • Ages 16-65
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Patients aged 16 to 65 years (included boundary value), of either sex;

  2. Patients with CMT1A (PMP22 duplication on chromosome 17p11.2) confirmed by genediagnosis; 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.

Exclusion

Exclusion Criteria:

  1. Being allergic to RS-baclofen, naltrexone HCL, D-sorbitol or any component inpxt3003 excipients or having other serious prior allergic reaction;

  2. Existence contraindications of baclofen, naltrexone or sorbitol, such as porphyria;

  3. Any other associated cause of peripheral neuropathy such as diabetes mellitus (including diabetes history and glycosylated hemoglobin >6.5%)

  4. Subjects with other neurological diseases affecting the evaluation of studytreatment;

  5. Patients with the score of ONLS score is 0;

  6. A history of unstable medical diseases with clinically significant unstable medicaldiseases (unstable angina pectoris, tumor, blood disease, hepatitis or liverfailure, renal failure, etc.) that may cause harm to the subjects participating inthis study in the past 1 year;

  7. Limb surgery had implemented within the first six months of randomization or will beplanned before the completion of the clinical trial;

  8. Hepatic or renal dysfunction:

  9. TBIL>1.5×ULN,ALT>3×ULN,AST>3×ULN;

  10. Cr>1.5×ULN;

  11. Syphilis antibody and HIV antibody positive subjects;

  12. Subjects with tumors indicated by chest radiograph or B-ultrasound;

  13. Subjects with alcohol dependence in recent 3 months;

  14. Females that are of childbearing potential, pregnant, or are breast-feeding;Subjectswho are unable to use appropriate contraceptives during the trial;

  15. Subjects with concomitant treatment 4 weeks before enrollment, including but notlimited to baclofen, naltrexone, sorbitol, opioids, vitamin C, levothyroxine andpotentially neurotoxic drugs (such as amiodarone and chloroquine);

  16. Subjects unable to complete the follow-up of study;

  17. Participated in another clinical trial and used the test drug within the last 30days;

  18. Different subjects from the same family and living in the same residence can onlyinclude one subject, so as to avoid treatment confusion, affect blind treatment andaffect the interpretation of the research results;

  19. Investigators affirm the compliance in a certain subject is poor or there are someother factors that are not suitable to participate in this clinical trial.

Study Design

Total Participants: 176
Treatment Group(s): 2
Primary Treatment: PXT3003 placebo
Phase: 3
Study Start date:
September 28, 2021
Estimated Completion Date:
March 18, 2024

Study Description

This multi-center, randomized, double-blind, placebo-controlled, Phase III clinical study is designed to evaluate PXT3003 versus placebo in subjects with genetically confirmed CMT1A of mild-to-moderate severity (CMTNS-V2 score >2 and ≤18) aged 16 to 65 years.

Genetically confirmed CMT1A subjects will be screened and randomized in a 1:1 ratio to receive either oral PXT3003 daily or matching placebo for 15 months. A total of approximately 176 subjects will be enrolled.

Visits will take place at Screening (up to -30 days), Baseline , and Months 3, 6, 9, 12, and 15.

Connect with a study center

  • Peking University Third Hospital

    Beijing,
    China

    Site Not Available

  • Xuanwu Hospital Capital Medical University

    Beijing,
    China

    Site Not Available

  • The First Bethune Hospital of Jilin University

    Changchun,
    China

    Site Not Available

  • The Third Xiangya Hospital of Central South University

    Changsha,
    China

    Site Not Available

  • The First Affiliated Hospital of Fujian Medical University

    Fuzhou,
    China

    Site Not Available

  • Nanfang Hospital Southern Medical University

    Guangzhou,
    China

    Site Not Available

  • The First Affiliated Hospital,Sun Yat-sen University

    Guangzhou,
    China

    Site Not Available

  • The Affiliated Hospital of Guizhou Medical University

    Guiyang,
    China

    Site Not Available

  • The Second Affiliated Hospital Zhejiang University School of Medicine

    Hangzhou,
    China

    Site Not Available

  • The First Affiliated Hospital of Harbin Medical University

    Harbin,
    China

    Site Not Available

  • The First Affiliated Hospital of Anhui Medical University

    Hefei,
    China

    Site Not Available

  • Inner Mongolia People's Hospital

    Hohhot,
    China

    Site Not Available

  • Qilu Hospital of Shandong University

    Jinan,
    China

    Site Not Available

  • The Second Affiliated Hospital of Nanchang University

    Nanchang,
    China

    Site Not Available

  • The Affiliated Hospital of Nanjing University Medical School

    Nanjing,
    China

    Site Not Available

  • Zhongda Hospital Southeast University

    Nanjing,
    China

    Site Not Available

  • Qilu Hospital of Shandong University(Qingdao)

    Qingdao,
    China

    Site Not Available

  • Shanghai Jiaotong University School of Medicine Ruijin Hospital

    Shanghai,
    China

    Site Not Available

  • The First Hospital of China Medical University

    Shenyang,
    China

    Site Not Available

  • The Second Hospital of Hebei Medical University

    Shijiazhuang,
    China

    Site Not Available

  • The First Affiliated Hospital of Soochow University

    Suzhou,
    China

    Site Not Available

  • Renmin Hospital of Wuhan University(Hubei General Hospital)

    Wuhan,
    China

    Site Not Available

  • The First Affiliated Hospital of Xi'an Jiaotong University

    Xi'an,
    China

    Site Not Available

  • Henan Provincial People's Hospital

    Zhengzhou,
    China

    Site Not Available

  • The First Affiliated Hospital of Zhengzhou University

    Zhengzhou,
    China

    Site Not Available

Map preview placeholder

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.