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

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.

Eligibility

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.

Conditions2

Interventions2

Browse More Trials