Evaluation of TQ-B3234 Capsules in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform Neurofibromas
Randomized, Double-Blind, Parallel-Controlled, Multicenter Phase III Clinical Trial Evaluating the Efficacy and Safety of TQ-B3234 Capsules Versus Placebo in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform Neurofibromas
1 other identifier
interventional
177
1 country
29
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Feb 2026
Typical duration for phase_3
29 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2026
CompletedFirst Submitted
Initial submission to the registry
February 3, 2026
CompletedFirst Posted
Study publicly available on registry
February 12, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2028
February 12, 2026
December 1, 2025
1.8 years
February 3, 2026
February 10, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
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)
Secondary Outcomes (15)
Investigator-Assessed ORR
From subject enrollment to the end of cycle 24 (each cycle is 28 days)
IRC/Investigator-Assessed DOR
From subject enrollment to the end of cycle 24 (each cycle is 28 days)
IRC/Investigator-Assessed disease control rate (DCR)
From subject enrollment to the end of cycle 24 (each cycle is 28 days)
IRC/Investigator-Assessed progression-free survival (PFS )
From subject enrollment to the end of cycle 24 (each cycle is 28 days)
IRC/Investigator-Assessed TTP
From subject enrollment to the end of cycle 24 (each cycle is 28 days)
- +10 more secondary outcomes
Study Arms (2)
TQ-B3234 capsules
EXPERIMENTALTQ-B3234 capsules: 50 mg once daily, one cycle every 4 weeks (28 days)
TQ-B3234 placebo
PLACEBO COMPARATORTQ-B3234 placebo: 0 mg once daily, one cycle every 4 weeks (28 days)
Interventions
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.
Eligibility Criteria
You may qualify if:
- The subject voluntarily joins this study, signs the informed consent form, and demonstrates good compliance.
- Age ≥18 years (calculated from the date of signing the informed consent form).
- Diagnosis of symptomatic, non-resectable neurofibromatosis type 1 (NF1)-associated plexiform neurofibroma (PN) requiring systemic therapy per investigator judgment.
- At least one measurable lesion with a dimension ≥3 cm.
- There should be no significant changes in the use of chronic neuropathic pain medications within 28 days prior to study enrollment.
- Eastern Cooperative Oncology Group (ECOG) performance status 0-1.
- Laboratory tests meet the protocol criteria.
- Women of childbearing potential must agree to use effective contraception during the study and for 6 months after study completion. A negative serum pregnancy test must be documented within 7 days prior to study enrollment. Men must agree to use effective contraception during the study and for 6 months after study completion.
You may not qualify if:
- Confirmed or suspected malignant glioma or malignant peripheral nerve sheath tumor (MPNST) (excluding low-grade glioma, optic nerve glioma not requiring systemic therapy or radiotherapy); histological confirmation may be required.
- History of or concurrent other malignancies within 5 years prior to first dosing.
- Multiple factors affecting oral drug absorption (e.g., dysphagia, chronic diarrhea, intestinal obstruction, major bowel resection).
- Adverse reactions from prior anti-tumor therapy not recovered to NCI CTCAE v6.0 grade ≤1, except grade 2 alopecia, grade 2 peripheral neuropathy, grade 2 anemia, non-clinically significant and asymptomatic laboratory abnormalities, and hypothyroidism stabilized by hormone replacement therapy.
- Major surgery, significant traumatic injury, or planned major surgery during the study within 4 weeks prior to first dosing; or presence of long-term non-healed wounds or fractures.
- History of arterial/venous thrombotic events (e.g., cerebrovascular accident including transient ischemic attack (TIA), deep vein thrombosis, pulmonary embolism) or other severe thromboembolic events within 6 months prior to first dosing.
- Active viral hepatitis with poor control.
- Active syphilis requiring treatment.
- Active tuberculosis, idiopathic pulmonary fibrosis, organizing pneumonia, drug-induced pneumonia, radiation pneumonitis requiring treatment, or clinically symptomatic active pneumonia.
- History of substance abuse that cannot be controlled or presence of psychiatric disorders.
- Planned or prior allogeneic bone marrow or solid organ transplantation.
- History of hepatic encephalopathy.
- History of or current retinal vein occlusion (RVO), retinal pigment epithelial detachment (RPED), central serous retinopathy (CSR), glaucoma, or other significant ocular abnormalities (e.g., intraocular pressure \>21mmHg).
- Inability to undergo MRI and/or presence of MRI contraindications.
- Major cardiovascular disease.
- +9 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (29)
The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui, 230000, China
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100032, China
Xuanwu Hospital Capital Medical University
Beijing, Beijing Municipality, 100032, China
Beijing Tiantan Hospital, Capital Medical University
Beijing, Beijing Municipality, 100070, China
Peking University Third Hospital
Beijing, Beijing Municipality, 100083, China
The Southwest Hospital of Amu
Chongqing, Chongqing Municipality, 400000, China
The First Affiliated Hospital of Fujan Medical University
Fuzhou, Fujian, 350004, China
Gansu Provincial Cancer Hospital
Lanzhou, Gansu, 730050, China
Sun Yat-Sen University Cancer Center
Guangzhou, Guangdong, 510060, China
Dermatology Hospital of Southern Medical University (Guangdong Provincial Dermatology Hospital / Guangdong Center for the Prevention and Control of Sexually Transmitted Diseases and Leprosy
Guangzhou, Guangdong, 510091, China
Guangxi Medical University Cancer Hospital （ Guangxi Cancer InstituteGuangxi Cancer Hospital & Medical University Oncology School & Cancer Center）
Nanning, Guangxi, 530021, China
Guizhou Provincial People's Hospital
Guiyang, Guizhou, 550002, China
The second hospital of Hebei medical university
Shijiazhuang, Hebei, 05000, China
The Second Affiliated Hospital of Henan University of Science and Technology
Luoyang, Henan, 471000, China
The First Affiliated Hospital Of Zhengzhou University
Zhengzhou, Henan, 450000, China
Renmin Hospital of Wuhan University(Hubei General Hospital)
Wuhan, Hubei, 42000, China
Hunan Children's Hospital
Changsha, Hunan, 410007, China
Nanjing Drum Tower Hospital
Nanjing, Jiangsu, 210008, China
The First Affiliated Hospital of China Medical University
Shenyang, Liaoning, 110801, China
Xijing hospital
Xi'an, Shaanxi, 710000, China
Qilu Hospital of Shandong University
Jinan, Shandong, 250012, China
Qilu Hospital of Shandong University
Jinan, Shandong, 250012, China
Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine
Shanghai, Shanghai Municipality, 200011, China
Shanxi Cancer Hospital
Taiyuan, Shanxi, 030000, China
West China Hospital, Sichuan University
Chengdu, Sichuan, 610041, China
Sichuan Academy of Medical Science&Sichuan Provincial People' Hospital
Chengdu, Sichuan, 610072, China
Tianjin Medical University Cancer Institute & Hospital
Tianjin, Tianjin Municipality, 300000, China
The First Affiliated Hospital Of Kunming Medical University
Kunming, Yunnan, 650000, China
Zhejiang Provincial People'S Hospital
Hangzhou, Zhejiang, 310014, China
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 3, 2026
First Posted
February 12, 2026
Study Start
February 1, 2026
Primary Completion (Estimated)
December 1, 2027
Study Completion (Estimated)
December 1, 2028
Last Updated
February 12, 2026
Record last verified: 2025-12