Evaluation of TQ-B3234 Capsules in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform Neurofibromas

May 22, 2026 updated by: Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

177

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
    • Anhui
      • Hefei, Anhui, China, 230000
        • Not yet recruiting
        • The First Affiliated Hospital of Anhui Medical University
        • Contact:
    • Beijing Municipality
      • Beijing, Beijing Municipality, China, 100032
        • Not yet recruiting
        • Peking Union Medical College Hospital
        • Contact:
      • Beijing, Beijing Municipality, China, 100032
        • Not yet recruiting
        • Xuanwu Hospital Capital Medical University
        • Contact:
      • Beijing, Beijing Municipality, China, 100070
        • Not yet recruiting
        • Beijing Tiantan Hospital, Capital Medical University
        • Contact:
      • Beijing, Beijing Municipality, China, 100083
        • Not yet recruiting
        • Peking University Third Hospital
        • Contact:
    • Chongqing Municipality
      • Chongqing, Chongqing Municipality, China, 400000
        • Recruiting
        • The Southwest Hospital of Amu
        • Contact:
    • Fujian
      • Fuzhou, Fujian, China, 350004
        • Not yet recruiting
        • The First Affiliated Hospital of Fujan Medical University
        • Contact:
    • Gansu
      • Lanzhou, Gansu, China, 730050
        • Not yet recruiting
        • Gansu Provincial Cancer Hospital
        • Contact:
    • Guangdong
      • Guangzhou, Guangdong, China, 510060
        • Not yet recruiting
        • Sun yat-sen University Cancer Center
        • Contact:
      • Guangzhou, Guangdong, China, 510091
        • 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
        • Contact:
    • Guangxi
      • Nanning, Guangxi, China, 530021
        • Not yet recruiting
        • Guangxi Medical University Cancer Hospital （ Guangxi Cancer InstituteGuangxi Cancer Hospital & Medical University Oncology School & Cancer Center）
        • Contact:
    • Guizhou
      • Guiyang, Guizhou, China, 550002
        • Not yet recruiting
        • Guizhou Provincial People's Hospital
        • Contact:
    • Hebei
      • Shijiazhuang, Hebei, China, 05000
        • Not yet recruiting
        • The Second Hospital of Hebei Medical University
        • Contact:
    • Henan
      • Luoyang, Henan, China, 471000
        • Not yet recruiting
        • The Second Affiliated Hospital of Henan University of Science and Technology
        • Contact:
      • Zhengzhou, Henan, China, 450000
        • Not yet recruiting
        • the First Affiliated Hospital of Zhengzhou University
        • Contact:
    • Hubei
      • Wuhan, Hubei, China, 42000
        • Not yet recruiting
        • Renmin Hospital of Wuhan University(Hubei General Hospital)
        • Contact:
    • Hunan
      • Changsha, Hunan, China, 410007
        • Not yet recruiting
        • Hunan Children's Hospital
        • Contact:
    • Jiangsu
      • Nanjing, Jiangsu, China, 210008
        • Not yet recruiting
        • Nanjing Drum Tower Hospital
        • Contact:
    • Liaoning
      • Shenyang, Liaoning, China, 110801
        • Not yet recruiting
        • The First Affiliated Hospital of China Medical University
        • Contact:
    • Shaanxi
      • Xi'an, Shaanxi, China, 710000
        • Not yet recruiting
        • Xijing Hospital
        • Contact:
    • Shandong
      • Jinan, Shandong, China, 250012
        • Not yet recruiting
        • Qilu Hospital of Shandong University
        • Contact:
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, China, 200011
        • Not yet recruiting
        • Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine
        • Contact:
    • Shanxi
      • Taiyuan, Shanxi, China, 030000
        • Recruiting
        • Shanxi Cancer Hospital
        • Contact:
    • Sichuan
      • Chengdu, Sichuan, China, 610041
        • Recruiting
        • West China Hospital, Sichuan University
        • Contact:
      • Chengdu, Sichuan, China, 610072
        • Not yet recruiting
        • Sichuan Academy of Medical Science&Sichuan Provincial People' Hospital
        • Contact:
    • Tianjin Municipality
      • Tianjin, Tianjin Municipality, China, 300000
        • Not yet recruiting
        • Tianjin Medical University Cancer Institute & Hospital
        • Contact:
    • Yunnan
      • Kunming, Yunnan, China, 650000
        • Not yet recruiting
        • The First Affiliated Hospital of Kunming Medical University
        • Contact:
    • Zhejiang
      • Hangzhou, Zhejiang, China, 310014
        • Not yet recruiting
        • Zhejiang Provincial People's Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.
  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: TQ-B3234 capsules
TQ-B3234 capsules: 50 mg once daily, one cycle every 4 weeks (28 days)
Drug: TQ-B3234 capsules
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.
Placebo Comparator: TQ-B3234 placebo
TQ-B3234 placebo: 0 mg once daily, one cycle every 4 weeks (28 days)
Drug: TQ-B3234 placebo
TQ-B3234 placebo without drug substance.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
IRC-Assessed Objective Response Rate (ORR)
Time Frame: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Investigator-Assessed ORR
Time Frame: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
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
Time Frame: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
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)
Time Frame: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
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 Frame: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
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 Frame: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
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 Frame: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
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
Time Frame: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
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)
Time Frame: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
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
Time Frame: From subject enrollment to the end of cycle 24 (each cycle is 28 days)
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)
Time Frame: 2 hours after administration
Maximum plasma drug concentration
2 hours after administration
Plasma concentration at steady state (Ctrough, SS)
Time Frame: 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)
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
Time Frame: From subject enrollment to the end of the 24th cycle (each cycle is 28 days)
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
Time Frame: From subject enrollment to the end of the 24th cycle (each cycle is 28 days)
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
Time Frame: From subject enrollment to the end of the 24th cycle (each cycle is 28 days)
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
Time Frame: From subject enrollment to the end of the 24th cycle (each cycle is 28 days)
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)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 31, 2026

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

December 1, 2028

Study Registration Dates

First Submitted

February 3, 2026

First Submitted That Met QC Criteria

February 10, 2026

First Posted (Actual)

February 12, 2026

Study Record Updates

Last Update Posted (Actual)

May 27, 2026

Last Update Submitted That Met QC Criteria

May 22, 2026

Last Verified

December 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TQ-B3234-III-01

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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