TQ-B3234 capsules compared with placebo for plexiform neurofibromas in neurofibromatosis type 1

Who can take part

• 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 take part

• 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)
• 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
• Planned or prior allogeneic bone marrow or solid organ transplantation
• Participation in and use of other PN clinical trial drugs within 4 weeks prior to first dosing
• 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
• Active viral hepatitis with poor control
• 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
• 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)
• 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
• Known hypersensitivity to study drug excipients
• Any other condition that, in the investigator's judgment, poses a serious risk to participant safety or interferes with study completion
• 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
• Active syphilis requiring treatment
• Inability to undergo MRI and/or presence of MRI contraindications
• Tumor-related symptoms and treatment
• Pregnant or lactating participants