Using Leflunomide to Treat MEN-1 Neuroendocrine Tumors
Phase II Clinical Study of Leflunomide in the Treatment of MEN-1 Neuroendocrine Tumor
This study is testing if leflunomide can be an effective second-line treatment for people with advanced neuroendocrine tumors related to Multiple Endocrine Neoplasia type 1 (MEN-1).
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | Cancer Institute and Hospital, Chinese Academy of Medical Sciences Academic / other |
| Drugs / interventions | methotrexate |
| Locations | 1 site (Beijing) |
| Trial ID | NCT06540937 on ClinicalTrials.gov |
What this trial studies
This phase II trial evaluates the effectiveness of leflunomide tablets as a second-line treatment for advanced neuroendocrine tumors associated with Multiple Endocrine Neoplasia type 1 (MEN-1). Eligible patients will take leflunomide daily and be monitored for disease progression and adverse reactions. The study includes regular follow-ups and assessments to track treatment effects and tumor status. Additionally, genetic testing will be conducted to analyze MEN-1 mutations in patients.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18-75 with unresectable neuroendocrine tumors and confirmed MEN-1 mutations who have failed first-line treatment.
Not a fit: Patients with resectable tumors or those who have not undergone first-line systemic therapy may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with advanced MEN-1 neuroendocrine tumors who have not responded to standard therapies.
How similar studies have performed: While this approach is novel for MEN-1 neuroendocrine tumors, similar studies using leflunomide in other cancer types have shown promising results.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age 18-75 years, gender unlimited * Pathological diagnosis of neuroendocrine tumor * MEN-1 germ line mutation (MEN-1 syndrome), or tumor with somatic MEN-1 mutation * Unresectable neuroendocrine tumors that have failed standard first-line systemic therapy * At least one evaluable lesion according to the RECIST criteria * ECOG physical condition score: 0-1 * Voluntarily joined the study, signed the informed consent, complied well, and cooperated with the follow-up. * Must meet the following requirements: Bone marrow: leukocyte \> 4.0×109/L, neutrophils ≥1.5×109/L, platelets ≥100×109/L, hemoglobin ≥10g/dL; Liver: total bilirubin \< 1.5 times the upper limit of normal, AST/ALT≤1.5 times the upper limit of normal value, Lactate dehydrogenase ≤1.5 times the upper limit of normal value, Alkaline phosphatase ≤5 times the upper limit of normal value; Kidney: serum creatinine ≤1.5 times the upper limit of normal value or creatinine clearance ≥60ml/min Exclusion Criteria: * Received any antitumor therapy within 4 weeks * Participated in or are participating in clinical trials of other drugs/therapies within 4 weeks prior to the initial use of the study drug * Underwent/received major surgery or has not recovered from the side effects of the surgery within 4 weeks prior to the initial use of the study drug * Subjects of other malignant tumors unless complete remission was achieved at least 2 years prior to study inclusion and not required other treatment during the study period * Persistent or active (serious) infection * Hypertension that is difficult to control with medication (continuous blood pressure is greater than 150/90 MMHG * Poorly controlled diabetes * Grade II/IV congestive heart failure or heart block * Within 6 months prior to use for the first time seen in the following situations: Deep vein thrombosis; Pulmonary embolism; Myocardial infarction; Serious arrhythmia; Instability or angina pectoris; Percutaneous coronary intervention; Acute coronary syndrome; Coronary artery bypass grafting * Be allergic to leflunomide tablets and their metabolites * Severe liver damage * Other severe acute or chronic medical conditions or abnormalities in laboratory tests that may increase the risks associated with study participation or may interfere with the interpretation of study results * Poor compliance, or other conditions unsuitable for participation in this trial
Where this trial is running
Beijing
- National Cancer Center/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College — Beijing, China (Recruiting)
Study contacts
- Study coordinator: Hong Zhao
- Email: pumc95zhao@126.com
- Phone: 010-87787100
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.