The Clinical Study of the Treatment of Patients With Type I Neurofibromatosis With Smetinib Hydrosulfate Capsule

Launched by SUN YAT-SEN MEMORIAL HOSPITAL OF SUN YAT-SEN UNIVERSITY · Sep 27, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a drug called Smetinib hydrosulfate capsule for patients with Type I Neurofibromatosis (NF1) who need surgery for their tumors. Neurofibromatosis is a condition that causes tumors to grow on nerves, and sometimes these tumors can come back after being surgically removed. The goal of the trial is to see if taking Smetinib after surgery can help shrink the tumors, reduce the chances of them coming back, and improve the overall effectiveness of treatment.

To be eligible for the trial, participants must be at least 18 years old and have a confirmed diagnosis of NF1 with certain characteristics, such as having multiple skin spots or specific types of tumors. They should also have a measurable tumor that is not invading critical areas like the brain or spine and must be in good overall health to tolerate the study medication. If you qualify and choose to participate, you will be monitored for how well the drug works in preventing tumor growth after your surgery. The study is not currently recruiting participants, but it aims to provide important information about a potential new treatment option for NF1 patients.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Age ≥18 years old
• • 2. According to the National Institutes of Health (NIH) updated diagnostic criteria for NF1 in 2021, 6 or more CALMs: d\>5 mm before puberty or d\>15 mm after puberty; 2 or more neurofibromas of any type or 1 plexiform neurofibroma;
• • ③ Freckles in the armpit or groin area;
• • ④ optic glioma (OPG);
• • ⑤ Two or more Lisch nodules were detected by slit-lamp, or two or more choroidal abnormalities were detected by optical coherence tomography (OCT)/ near-infrared (NIR) imaging;
• • ⑥ Characteristic bone lesions, such as sphenoid dysplasia, anterolateral tibial curvature; Pathogenic heterozygote NF1 variant with 50% allele variant fraction in normal tissues (such as white blood cells); NF1 is diagnosed in persons who have no history of parental disease and meet 2 or more clinical characteristics Individuals with a history of parental disease who meet one or more clinical characteristics may be diagnosed with NF1
• • 3. Before admission, the head and neck surgeon conducted pathological biopsy of solid tumors, confirmed pathological diagnosis and eliminated malignant peripheral schwannoma (MPNST).
• • 4. There was at least one measurable tumor lesion according to the solid tumor efficacy evaluation criteria RECIST 1.1
• • 5. The tumor did not invade the brain, spine and other important organs, there are indications of surgical resection and surgical treatment
• • 6. The performance of the Eastern Cooperative Oncology Group (ECOG) was 0-1
• • 7. Blood routine: white blood cell count (WBC) ≥3.0×109/L; Absolute neutrophil count (ANC) ≥ 1.5×109/L; Platelet (PLT) ≥ 100×109/L; Hemoglobin level (HGB) ≥ 9.0 g/dL (7 days without corresponding supportive treatment, such as blood transfusion and increased white blood cells).
• • 8. Liver function: the patient's aspartate aminotransferase (AST) and alanine aminotransferase (ALT) were less than 2.5 times the upper limit of reference value (ULN); Albumin (ALB) ≥ 30 g/L.
• • 9. Renal function: serum creatinine ≤1.5 times ULN or creatinine clearance (CrCl) ≥ 50mL/min (using Cockcroft/Gault formula); Urinary protein (UPRO) \< (++), or 24-hour urinary protein volume \< 1.0 g.
• • 10. Cardiac function: creatine phosphokinase ≤200U/L, left ventricular ejection fraction (LVEF) ≥50%;
• • 11. Have not participated in other clinical trials within the past 30 days;
• • 12. Patients who voluntarily participate in the project and sign informed consent.
• Exclusion Criteria:
• • 1. The patient had abnormal blood indexes and abnormal liver, kidney and heart function, and could not tolerate the clinical study process after multidisciplinary consultation and evaluation
• • 2. The patient has become malignant peripheral schwannoma (MPNST) or is accompanied by other malignant tumors, heart disease and other serious complications, or has previously received chemotherapy, radiotherapy and other anti-tumor therapy
• • 3. Unable to complete the entire clinical study due to personal, social and economic reasons
• • 4. there is a serious systemic disease in the past and the disease cannot be cured or controlled by medicine at present
• • 5. Pregnant patients