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This study aims to demonstrate that in subjects with symptomatic, inoperable plexiform neurofibromas associated with neurofibromatosis type 1, TQ-B3234 capsules significantly improve the objective response rate at Week 24 compared to placebo.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TQ-B3234 capsules | Experimental | TQ-B3234 capsules: 50 mg once daily, one cycle every 4 weeks (28 days) |
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| TQ-B3234 placebo | Placebo Comparator | TQ-B3234 placebo: 0 mg once daily, one cycle every 4 weeks (28 days) |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TQ-B3234 capsules | Drug | TQ-B3234 is an antitumor molecular targeted drug, a selective mitogen-activated protein kinase 1 and 2 (MEK1/2) inhibitor. It primarily inhibits the mitogen-activated protein kinase (MEK) protein (an upstream regulator of the extracellular signal-regulated kinase (ERK) pathway), thereby affecting the mitogen-activated protein kinase (MAPK) pathway and suppressing cell proliferation. MEK inhibitors are recognized to play a significant role in the pathogenesis of plexiform neurofibromas associated with neurofibromatosis type 1. |
| Measure | Description | Time Frame |
|---|---|---|
| IRC-Assessed Objective Response Rate (ORR) | Percentage of subjects achieving complete response (CR) or partial response (PR) as assessed by independent review committee (IRC) per REiNS criteria at the end of Cycle 24. | From subject enrollment to the end of cycle 24 (each cycle is 28 days) |
| Measure | Description | Time Frame |
|---|---|---|
| Investigator-Assessed ORR | Percentage of subjects achieving complete response (CR) or partial response (PR) as assessed by investigators per REiNS criteria. | From subject enrollment to the end of cycle 24 (each cycle is 28 days) |
| IRC/Investigator-Assessed DOR |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Qingfeng Li, Doctor | Contact | 021 53315108 | dr.liqingfeng@shsmu.edu.cn |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The First Affiliated Hospital of Anhui Medical University | Not yet recruiting | Hefei | Anhui | 230000 | China |
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| TQ-B3234 placebo | Drug | TQ-B3234 placebo without drug substance. |
|
Duration of response (DOR), defined as the time from first documented confirmed objective response to disease progression or death from any cause (whichever occurs first), as determined by IRC/investigators per REiNS criteria. |
| From subject enrollment to the end of cycle 24 (each cycle is 28 days) |
| IRC/Investigator-Assessed disease control rate (DCR) | Percentage of subjects achieving complete response (CR), partial response (PR), or stable disease (SD) as determined by IRC/investigators per REiNS criteria. | From subject enrollment to the end of cycle 24 (each cycle is 28 days) |
| IRC/Investigator-Assessed progression-free survival (PFS ) | Time from randomization to first disease progression or death from any cause (whichever occurs first), as determined by IRC/investigators per REiNS criteria. | From subject enrollment to the end of cycle 24 (each cycle is 28 days) |
| IRC/Investigator-Assessed TTP | Time from randomization to first disease progression (TTP), as assessed by IRC/investigators per REiNS criteria. | From subject enrollment to the end of cycle 24 (each cycle is 28 days) |
| IRC/Investigator-Assessed time to response (TTR) | Time from randomization to first objective response, as determined by IRC/investigators per REiNS criteria. | From subject enrollment to the end of cycle 24 (each cycle is 28 days) |
| Tumor response in subject | Difference in the best percentage change from baseline in target PN volume between groups, as assessed by IRC/investigators. | From subject enrollment to the end of cycle 24 (each cycle is 28 days) |
| Number of subjects with incidence and severity of adverse events (AEs) | Incidence and severity of adverse events from subject enrollment to the end of cycle 24. | From subject enrollment to the end of cycle 24 (each cycle is 28 days) |
| Patient-reported outcomes | Patient self-assessment results from subject enrollment to the end of cycle 24. | From subject enrollment to the end of cycle 24 (each cycle is 28 days) |
| Peak concentration (Cmax) | Maximum plasma drug concentration | 2 hours after administration |
| Plasma concentration at steady state (Ctrough, SS) | The pre-dose concentration observed when the drug has reached steady state with repeated dosing, indicating baseline exposure and accumulation. | Cycle 1 day 28: pre-dose, Cycle 2 day 28 pre-dose, Cycle 3 day 28: pre-dose, Cycle 6 day 28: pre-dose. (each cycle is 28 days) |
| Effects on pain score in subjects | Questionnaire: Numerical Rating Scale-11 (NRS-11); The line segments below all have numbers from 0 to 10, where 0 means no pain and 10 is the worst pain you can imagine. | From subject enrollment to the end of the 24th cycle (each cycle is 28 days) |
| Effects on pain interference index in subjects | Questionnaire: pain interference index; Please answer each one by circling a number from 0 to 6, where 0 means none at all and 6 means completely | From subject enrollment to the end of the 24th cycle (each cycle is 28 days) |
| Effects on subjects' general quality of life | Questionnaire: Functional Assessment of Cancer Therapy - General (FACT-G); the scores from all items across its four domains are summed to obtain a total score, which ranges from 0 to 108 points. | From subject enrollment to the end of the 24th cycle (each cycle is 28 days) |
| Effects on subjects; disease-specific quality of life | Questionnaire: PedsQL NF1 Module; It includes an Adult version (>25 years) and a Young Adult version (18-25 years), with 104 items, respectively. | From subject enrollment to the end of the 24th cycle (each cycle is 28 days) |
| Peking Union Medical College Hospital | Not yet recruiting | Beijing | Beijing Municipality | 100032 | China |
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| Xuanwu Hospital Capital Medical University | Not yet recruiting | Beijing | Beijing Municipality | 100032 | China |
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| Beijing Tiantan Hospital, Capital Medical University | Not yet recruiting | Beijing | Beijing Municipality | 100070 | China |
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| Peking University Third Hospital | Not yet recruiting | Beijing | Beijing Municipality | 100083 | China |
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| The Southwest Hospital of Amu | Recruiting | Chongqing | Chongqing Municipality | 400000 | China |
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| The First Affiliated Hospital of Fujan Medical University | Not yet recruiting | Fuzhou | Fujian | 350004 | China |
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| Gansu Provincial Cancer Hospital | Not yet recruiting | Lanzhou | Gansu | 730050 | China |
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| Sun Yat-Sen University Cancer Center | Not yet recruiting | Guangzhou | Guangdong | 510060 | China |
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| Dermatology Hospital of Southern Medical University (Guangdong Provincial Dermatology Hospital / Guangdong Center for the Prevention and Control of Sexually Transmitted Diseases and Leprosy | Not yet recruiting | Guangzhou | Guangdong | 510091 | China |
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| Guangxi Medical University Cancer Hospital ( Guangxi Cancer InstituteGuangxi Cancer Hospital & Medical University Oncology School & Cancer Center) | Not yet recruiting | Nanning | Guangxi | 530021 | China |
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| Guizhou Provincial People's Hospital | Not yet recruiting | Guiyang | Guizhou | 550002 | China |
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| The second hospital of Hebei medical university | Not yet recruiting | Shijiazhuang | Hebei | 05000 | China |
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| The Second Affiliated Hospital of Henan University of Science and Technology | Not yet recruiting | Luoyang | Henan | 471000 | China |
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| The First Affiliated Hospital Of Zhengzhou University | Not yet recruiting | Zhengzhou | Henan | 450000 | China |
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| Renmin Hospital of Wuhan University(Hubei General Hospital) | Not yet recruiting | Wuhan | Hubei | 42000 | China |
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| Hunan Children's Hospital | Not yet recruiting | Changsha | Hunan | 410007 | China |
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| Nanjing Drum Tower Hospital | Not yet recruiting | Nanjing | Jiangsu | 210008 | China |
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| The First Affiliated Hospital of China Medical University | Not yet recruiting | Shenyang | Liaoning | 110801 | China |
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| Xijing hospital | Not yet recruiting | Xi'an | Shaanxi | 710000 | China |
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| Qilu Hospital of Shandong University | Not yet recruiting | Jinan | Shandong | 250012 | China |
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| Qilu Hospital of Shandong University | Not yet recruiting | Jinan | Shandong | 250012 | China |
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| Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine | Not yet recruiting | Shanghai | Shanghai Municipality | 200011 | China |
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| Shanxi Cancer Hospital | Recruiting | Taiyuan | Shanxi | 030000 | China |
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| West China Hospital, Sichuan University | Recruiting | Chengdu | Sichuan | 610041 | China |
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| Sichuan Academy of Medical Science&Sichuan Provincial People' Hospital | Not yet recruiting | Chengdu | Sichuan | 610072 | China |
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| Tianjin Medical University Cancer Institute & Hospital | Not yet recruiting | Tianjin | Tianjin Municipality | 300000 | China |
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| The First Affiliated Hospital Of Kunming Medical University | Not yet recruiting | Kunming | Yunnan | 650000 | China |
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| Zhejiang Provincial People'S Hospital | Not yet recruiting | Hangzhou | Zhejiang | 310014 | China |
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| ID | Term |
|---|---|
| D018318 | Neurofibroma, Plexiform |
| ID | Term |
|---|---|
| D009455 | Neurofibroma |
| D018317 | Nerve Sheath Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D010524 | Peripheral Nervous System Neoplasms |
| D009423 | Nervous System Neoplasms |
| D009422 | Nervous System Diseases |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
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