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

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 will see if oral TQ-B3234 capsules can shrink or reduce symptoms from inoperable plexiform neurofibromas in adults with neurofibromatosis type 1 by Week 24 compared with placebo.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 177 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Chia Tai Tianqing Pharmaceutical Group Co., Ltd. (Industry-sponsored)
• Drugs / interventions: radiation
• Locations: 29 sites (Hefei, Anhui and 28 other locations)
• Trial ID: NCT07407803 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Adults aged 18 or older with symptomatic, non-resectable NF1-associated plexiform neurofibromas, at least one measurable lesion ≥3 cm, ECOG 0–1, and stable neuropathic pain medication use are ideal candidates.

Not a fit: Children, patients with surgically removable tumors, those with poor performance status or disqualifying lab abnormalities, or who cannot maintain stable pain medications or contraception may not benefit from participation.

Why it matters

Potential benefit: If successful, TQ-B3234 could shrink plexiform neurofibromas and reduce related symptoms, providing a non-surgical treatment option for adults with inoperable NF1 tumors.

How similar studies have performed: Targeted medical treatment of plexiform neurofibromas has precedent—MEK inhibitors like selumetinib have shrunk PNs in pediatric patients—while TQ-B3234 represents a novel agent being tested in adults.

Eligibility criteria

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 this trial is running

Hefei, Anhui and 28 other locations

• 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)
• Beijing Tiantan Hospital, Capital Medical University — Beijing, Beijing Municipality, China (Not_yet_recruiting)
• Peking University Third Hospital — Beijing, Beijing Municipality, China (Not_yet_recruiting)
• The Southwest Hospital of Amu — Chongqing, Chongqing Municipality, China (Recruiting)
• The First Affiliated Hospital of Fujan Medical University — Fuzhou, Fujian, China (Not_yet_recruiting)
• Gansu Provincial Cancer Hospital — Lanzhou, Gansu, China (Not_yet_recruiting)
• Sun Yat-Sen University Cancer Center — Guangzhou, Guangdong, China (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 — Guangzhou, Guangdong, China (Not_yet_recruiting)
• Guangxi Medical University Cancer Hospital （ Guangxi Cancer InstituteGuangxi Cancer Hospital & Medical University Oncology School & Cancer Center） — Nanning, Guangxi, China (Not_yet_recruiting)
• Guizhou Provincial People's Hospital — Guiyang, Guizhou, China (Not_yet_recruiting)
• The second hospital of Hebei medical university — Shijiazhuang, Hebei, China (Not_yet_recruiting)
• The Second Affiliated Hospital of Henan University of Science and Technology — Luoyang, Henan, China (Not_yet_recruiting)
• The First Affiliated Hospital Of Zhengzhou University — Zhengzhou, Henan, China (Not_yet_recruiting)
• Renmin Hospital of Wuhan University(Hubei General Hospital) — Wuhan, Hubei, China (Not_yet_recruiting)
• Hunan Children's Hospital — Changsha, Hunan, China (Not_yet_recruiting)
• Nanjing Drum Tower Hospital — Nanjing, Jiangsu, China (Not_yet_recruiting)
• The First Affiliated Hospital of China Medical University — Shenyang, Liaoning, China (Not_yet_recruiting)
• Xijing hospital — Xi'an, Shaanxi, China (Not_yet_recruiting)
• Qilu Hospital of Shandong University — Jinan, Shandong, China (Not_yet_recruiting)
• Qilu Hospital of Shandong University — Jinan, Shandong, China (Not_yet_recruiting)
• Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine — Shanghai, Shanghai Municipality, China (Not_yet_recruiting)
• Shanxi Cancer Hospital — Taiyuan, Shanxi, China (Recruiting)
• West China Hospital, Sichuan University — Chengdu, Sichuan, China (Recruiting)
• Sichuan Academy of Medical Science&Sichuan Provincial People' Hospital — Chengdu, Sichuan, China (Not_yet_recruiting)
• Tianjin Medical University Cancer Institute & Hospital — Tianjin, Tianjin Municipality, China (Not_yet_recruiting)
• The First Affiliated Hospital Of Kunming Medical University — Kunming, Yunnan, China (Not_yet_recruiting)
• Zhejiang Provincial People'S Hospital — Hangzhou, Zhejiang, China (Not_yet_recruiting)

Study contacts

• Study coordinator: Qingfeng Li, Doctor

Conditions Plexiform Neurofibroma