Clinical Study on the Treatment of Type I Neurofibromatosis With Smeitinib 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 new treatment for people with Type I Neurofibromatosis (NF1), a condition that can cause tumors to grow on nerves. The treatment being tested is a capsule called Smetinib, which aims to shrink these tumors and possibly make surgery safer and more effective for patients who need it. Currently, there are no good drug options for this condition, and surgery can often lead to the tumors coming back. The goal of this study is to see if Smetinib can help reduce the size of tumors and improve surgery outcomes.

To be eligible for this trial, participants must be at least 18 years old and meet specific criteria that show they have NF1, such as having certain types of skin spots and tumors. Patients should also have measurable tumors that are currently not suitable for surgery. Throughout the trial, participants will receive Smetinib and will be monitored to see how well it works in shrinking their tumors and improving their overall health. It’s important to note that this study is not yet recruiting participants, and those interested should consult their healthcare provider for more information.

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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 invaded the brain, spine and other important organs, no indication of surgical resection
• • 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. Patients have malignant peripheral schwannoma (MPNST) or other malignant tumors, heart disease and other serious complications, or have previously undergone anti-tumor therapy such as surgery, chemotherapy, radiotherapy
• • 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