Plexiform NeurofibromaPhase 3NCT07407803
Evaluation of TQ-B3234 Capsules in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform Neurofibromas
A Phase 3 study testing TQ-B3234 capsules in people with Plexiform Neurofibroma, run by Chia Tai Tianqing Pharmaceutical Group Co., Ltd. at 29 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Plexiform Neurofibroma
- What is being tested?
- TQ-B3234 capsules
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 177 participants
- Where
- 29 sitesChina
- Study dates
- 31 Mar 2026to Dec 2028
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
- Active or uncontrolled severe infection.
- Renal failure requiring hemodialysis or peritoneal dialysis.
- History of immunodeficiency, including HIV-positive or other acquired/congenital immunodeficiency diseases.
- History of epilepsy.
- Tumor-related symptoms and treatment.
- Known hypersensitivity to study drug excipients.
- Participation in and use of other PN clinical trial drugs within 4 weeks prior to first dosing.
- Pregnant or lactating participants.
- Any other condition that, in the investigator's judgment, poses a serious risk to participant safety or interferes with study completion.
Trial locations and contacts
29 sites in 1 country. Contact the study team or a site near you to ask about taking part.
29 sites in 1 country
China29 sites, 29 recruiting
- Not yet recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100032
Xiao Long, Doctor, 13810193175, pumclongxiao@126.com
- Not yet recruiting
Xuanwu Hospital Capital Medical University
Beijing, Beijing Municipality, 100032
Qingtang Lin, Doctor, 15801588169, kingsang2002@hotmail.com
- Not yet recruiting
Beijing Tiantan Hospital, Capital Medical University
Beijing, Beijing Municipality, 100070
Bo Wang, Doctor, 13811713730, docking@163.com
- Not yet recruiting
Peking University Third Hospital
Beijing, Beijing Municipality, 100083
Liping Wang, Doctor, 13810002694, dianen@126.com
- Not yet recruiting
Hunan Children's Hospital
Changsha, Hunan, 410007
Liwen Wu, Doctor, 137872458, hnsetyysjnk168@163.com
- Recruiting
West China Hospital, Sichuan University
Chengdu, Sichuan, 610041
Yu Cao, Doctor, 18980601184, caoyu@wchscu.cn
- Not yet recruiting
Sichuan Academy of Medical Science&Sichuan Provincial People' Hospital
Chengdu, Sichuan, 610072
Yaqiu Wu, Bachelor, 13551213719, wuyaqiu829@163.om
- Recruiting
The Southwest Hospital of Amu
Chongqing, Chongqing Municipality, 400000
Jiaping Zhang, Doctor, 13452964445, japzhang@tmmu.edu.cn
- Not yet recruiting
The First Affiliated Hospital of Fujan Medical University
Fuzhou, Fujian, 350004
Yuanxiang Lin, Doctor, 13906918894, lyx99070@163.com
- Not yet recruiting
Sun Yat-Sen University Cancer Center
Guangzhou, Guangdong, 510060
Zhongping Chen, Doctor, 13500002457, chenzhp@sysucc.org.cn
- Not yet recruiting
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
Zhimiao Lin, Doctor, 13681438841, zhimiaolin@126.com
- Not yet recruiting
Guizhou Provincial People's Hospital
Guiyang, Guizhou, 550002
DEYI ZHENG, Doctor, 139 8482 6262, deyizheng@126.com
- Not yet recruiting
Zhejiang Provincial People'S Hospital
Hangzhou, Zhejiang, 310014
Shuofan Wu, Doctor, 13857121888, sufanwu@163.com
- Not yet recruiting
The First Affiliated Hospital of Anhui Medical University
Hefei, Anhui, 230000
Beicheng Sun, Doctor, 13776413940, sunbc0207@163.com
- Not yet recruiting
Qilu Hospital of Shandong University
Jinan, Shandong, 250012
Shilei Ni, Doctor, 13869196590, nishilei@sdu.edu.cn
- Not yet recruiting
Qilu Hospital of Shandong University
Jinan, Shandong, 250012
Aijun Zhang, Doctor, 18560086212, zhangaijun@sdu.edu.cn
- Not yet recruiting
The First Affiliated Hospital Of Kunming Medical University
Kunming, Yunnan, 650000
YANG TANG, Doctor, 13518780688, drtangyang@126.com
- Not yet recruiting
Gansu Provincial Cancer Hospital
Lanzhou, Gansu, 730050
Jing Li, Bachelor, 13321229317, 1368406388@qq.com
- Not yet recruiting
The Second Affiliated Hospital of Henan University of Science and Technology
Luoyang, Henan, 471000
Ying Yao, Doctor, 15537990507, 15537990507@163.com
- Not yet recruiting
Nanjing Drum Tower Hospital
Nanjing, Jiangsu, 210008
Zezhang Zhu, Doctor, 13951916106, zhuzezhang@126.com
- Not yet recruiting
Guangxi Medical University Cancer Hospital （ Guangxi Cancer InstituteGuangxi Cancer Hospital & Medical University Oncology School & Cancer Center）
Nanning, Guangxi, 530021
Ligen Mo, Doctor, 13807816094, ligenmo@163.com
- Not yet recruiting
Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine
Shanghai, Shanghai Municipality, 200011
Qingfeng Li, Doctor, 021 53315108, dr.liqingfeng@shsmu.edu.cn
- Not yet recruiting
The First Affiliated Hospital of China Medical University
Shenyang, Liaoning, 110801
Xinghua Gao, Doctor, 13940152467, gaobarry@hotmail.com
- Not yet recruiting
The second hospital of Hebei medical university
Shijiazhuang, Hebei, 05000
Yanling Li, Doctor, 15130119920, lyldoctor@sina.com
- Recruiting
Shanxi Cancer Hospital
Taiyuan, Shanxi, 030000
Jianghua Wei, Master, 13700503026, 3793556386@qq.com
- Not yet recruiting
Tianjin Medical University Cancer Institute & Hospital
Tianjin, Tianjin Municipality, 300000
Jilong Yang, Doctor, 18622221626, yangjilong@tjmuch.com
- Not yet recruiting
Renmin Hospital of Wuhan University(Hubei General Hospital)
Wuhan, Hubei, 42000
Shan Jiang, Doctor, 13659892065, Jiangshan794101@126.com
- Not yet recruiting
Xijing hospital
Xi'an, Shaanxi, 710000
Baoqiang Song, Doctor, 18829025566, songbq1@163.com
- Not yet recruiting
The First Affiliated Hospital Of Zhengzhou University
Zhengzhou, Henan, 450000
Guangshuai Li, Doctor, 13838062386, liguangshuai@zzu.edu.cn
About this study
Official title: 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
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.
- Conditions
- Plexiform Neurofibroma
- Being tested
- TQ-B3234 capsules, TQ-B3234 placebo
- Medical subject headings
- Neurofibroma, Plexiform
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TQ-B3234 capsulesDrug
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.
TQ-B3234 placeboDrug
TQ-B3234 placebo without drug substance.
| Study group | Role | What they receive |
|---|---|---|
| TQ-B3234 capsules | Experimental | TQ-B3234 capsules: 50 mg once daily, one cycle every 4 weeks (28 days) Receives: Drug: TQ-B3234 capsules |
| TQ-B3234 placebo | Placebo comparator | TQ-B3234 placebo: 0 mg once daily, one cycle every 4 weeks (28 days) Receives: Drug: TQ-B3234 placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
Secondary outcomes (15)
Investigator-Assessed ORR
Percentage of subjects achieving complete response (CR) or partial response (PR) as assessed by investigators per REiNS criteria.
Measured: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
IRC/Investigator-Assessed DOR
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
Peak concentration (Cmax)
Maximum plasma drug concentration
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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.
Measured: From subject enrollment to the end of the 24th cycle (each cycle is 28 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2026
- Study start31 Mar 2026
- Primary completionDec 2027 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Plexiform NeurofibromaStudies of the same condition, open and under way.
- More trials run by Chia Tai Tianqing Pharmaceutical Group Co., Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 177 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07407803
- Sponsor's protocol ID
- TQ-B3234-III-01
- Status last verified
- Dec 2025
- First submitted
- 3 Feb 2026
- Last updated
- 27 May 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No