NCT07407803招募中3 期
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
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 177
- 试验地点
- 28
- 主要终点
- IRC-Assessed Objective Response Rate (ORR)
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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-
- •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.
排除标准
- •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.
研究组 & 干预措施
TQ-B3234 capsules
Experimental
TQ-B3234 capsules: 50 mg once daily, one cycle every 4 weeks (28 days)
干预措施: TQ-B3234 capsules (Drug)
TQ-B3234 placebo
Placebo Comparator
TQ-B3234 placebo: 0 mg once daily, one cycle every 4 weeks (28 days)
干预措施: TQ-B3234 placebo (Drug)
结局指标
主要结局
IRC-Assessed Objective Response Rate (ORR)
时间窗: 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.
次要结局
- IRC/Investigator-Assessed TTP(From subject enrollment to the end of cycle 24 (each cycle is 28 days))
- IRC/Investigator-Assessed time to response (TTR)(From subject enrollment to the end of cycle 24 (each cycle is 28 days))
- Tumor response in subject(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)(From subject enrollment to the end of cycle 24 (each cycle is 28 days))
- Patient-reported outcomes(From subject enrollment to the end of cycle 24 (each cycle is 28 days))
- Peak concentration (Cmax)(2 hours after administration)
- Plasma concentration at steady state (Ctrough, SS)(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(From subject enrollment to the end of the 24th cycle (each cycle is 28 days))
- Effects on pain interference index in subjects(From subject enrollment to the end of the 24th cycle (each cycle is 28 days))
- IRC/Investigator-Assessed DOR(From subject enrollment to the end of cycle 24 (each cycle is 28 days))
- IRC/Investigator-Assessed disease control rate (DCR)(From subject enrollment to the end of cycle 24 (each cycle is 28 days))
- IRC/Investigator-Assessed progression-free survival (PFS )(From subject enrollment to the end of cycle 24 (each cycle is 28 days))
- Investigator-Assessed ORR(From subject enrollment to the end of cycle 24 (each cycle is 28 days))
- Effects on subjects' general quality of life(From subject enrollment to the end of the 24th cycle (each cycle is 28 days))
- Effects on subjects; disease-specific quality of life(From subject enrollment to the end of the 24th cycle (each cycle is 28 days))
研究者
研究点 (28)
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