TQ-B3234 capsules for adults with symptomatic, inoperable NF1 plexiform neurofibromas

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 3 interventional trial comparing oral TQ-B3234 capsules to matched placebo in adults with symptomatic, non-resectable NF1-associated plexiform neurofibromas. Participants must have at least one measurable lesion ≥3 cm and meet performance status and laboratory criteria, with stable use of chronic neuropathic pain medications before enrollment. Subjects are randomized to receive TQ-B3234 or placebo and are followed through Week 24 for objective tumor response and safety monitoring. Study sites are located at three major hospitals in China and require contraception for participants of childbearing potential.

Why it matters

Who can join: the full rules

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.

Where it's running

• The Southwest Hospital of Amu
  Chongqing, Chongqing Municipality, China Recruiting
• Shanxi Cancer Hospital
  Taiyuan, Shanxi, China Recruiting
• West China Hospital, Sichuan University
  Chengdu, Sichuan, China Recruiting
• The First Affiliated Hospital of Anhui Medical University
  Hefei, Anhui, China Not yet recruiting
• Peking Union Medical College Hospital
  Beijing, Beijing Municipality, China Not yet recruiting
• Xuanwu Hospital Capital Medical University
  Beijing, Beijing Municipality, China Not yet recruiting

Study contacts

• Study coordinator: Qingfeng Li, Doctor
• Email: dr.liqingfeng@shsmu.edu.cn
• Phone: 021 53315108

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Combination therapy to shrink plexiform neurofibromas in NF1 patients

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.