A Study to Assess the Efficacy and Safety of PXT3003 in Charcot-Marie-Tooth Type 1A
NCT ID: NCT05092841
Last Updated: 2025-01-09
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE3
176 participants
INTERVENTIONAL
2021-09-28
2024-03-18
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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PXT3003
Liquid oral solution, 10 mL twice a day, morning and evening with food
PXT3003
Patients will be administered PXT3003 twice daily (bid) at 10mL.
PXT3003 Placebo
Liquid oral solution, 10 mL twice a day, morning and evening with food
PXT3003 placebo
Patients will be administered PXT3003 placebo twice daily (bid) at 10mL.
Interventions
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PXT3003
Patients will be administered PXT3003 twice daily (bid) at 10mL.
PXT3003 placebo
Patients will be administered PXT3003 placebo twice daily (bid) at 10mL.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. 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.
Exclusion Criteria
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 study treatment;
5. Patients with the score of ONLS score is 0;
6. 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;
7. Limb surgery had implemented within the first six months of randomization or will be planned before the completion of the clinical trial;
8. Hepatic or renal dysfunction:
1. TBIL\>1.5×ULN,ALT\>3×ULN,AST\>3×ULN;
2. Cr\>1.5×ULN;
9. Syphilis antibody and HIV antibody positive subjects;
10. Subjects with tumors indicated by chest radiograph or B-ultrasound;
11. Subjects with alcohol dependence in recent 3 months;
12. Females that are of childbearing potential, pregnant, or are breast-feeding;Subjects who are unable to use appropriate contraceptives during the trial;
13. 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);
14. Subjects unable to complete the follow-up of study;
15. Participated in another clinical trial and used the test drug within the last 30 days;
16. 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;
17. 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.
16 Years
65 Years
ALL
No
Sponsors
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Tasly GeneNet Pharmaceuticals Co., Ltd
INDUSTRY
Responsible Party
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Principal Investigators
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Rui Liu
Role: STUDY_DIRECTOR
Tasly Group,Co.Ltd.
Locations
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Peking University Third Hospital
Beijing, , China
Xuanwu Hospital Capital Medical University
Beijing, , China
The First Bethune Hospital of Jilin University
Changchun, , China
The Third Xiangya Hospital of Central South University
Changsha, , China
The First Affiliated Hospital of Fujian Medical University
Fuzhou, , China
Nanfang Hospital Southern Medical University
Guangzhou, , China
The First Affiliated Hospital，Sun Yat-sen University
Guangzhou, , China
The Affiliated Hospital of Guizhou Medical University
Guiyang, , China
The Second Affiliated Hospital Zhejiang University School of Medicine
Hangzhou, , China
The First Affiliated Hospital of Harbin Medical University
Harbin, , China
The First Affiliated Hospital of Anhui Medical University
Hefei, , China
Inner Mongolia People's Hospital
Hohhot, , China
Qilu Hospital of Shandong University
Jinan, , China
The Second Affiliated Hospital of Nanchang University
Nanchang, , China
The Affiliated Hospital of Nanjing University Medical School
Nanjing, , China
Zhongda Hospital Southeast University
Nanjing, , China
Qilu Hospital of Shandong University（Qingdao）
Qingdao, , China
Shanghai Jiaotong University School of Medicine Ruijin Hospital
Shanghai, , China
The First Hospital of China Medical University
Shenyang, , China
The Second Hospital of Hebei Medical University
Shijiazhuang, , China
The First Affiliated Hospital of Soochow University
Suzhou, , China
Renmin Hospital of Wuhan University（Hubei General Hospital）
Wuhan, , China
The First Affiliated Hospital of Xi'an Jiaotong University
Xi'an, , China
Henan Provincial People's Hospital
Zhengzhou, , China
The First Affiliated Hospital of Zhengzhou University
Zhengzhou, , China
Countries
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Other Identifiers
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TSL-CM-PXT3003-Ⅲ
Identifier Type: -
Identifier Source: org_study_id
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