Phase II Clinical Study of Leflunomide in the Treatment of MEN-1 Neuroendocrine Tumor

NCT06540937 | Recruiting Phase 2

• 1 other identifier: NCC2409
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

This phase II trial studies aim to evaluate the initial efficacy of leflunomide tablets in second-line treatment of advanced MEN-1 neuroendocrine tumors, and to provide evidence for phase III clinical trials.

Conditions

Neuroendocrine Tumors

Keywords

Multiple Endocrine Neoplasia; Leflunomide

Outcome Measures

Primary Outcomes (2)

• Overall response rate (ORR) assessed by RECIST 1.1

  ORR is defined as the percentage of patients with a complete response (CR) or partial response (PR) according to RECIST v1.1.

  up to 36 weeks

• Progression-free survival (PFS)

  PFS is defined as the duration from the date of randomization to the onset of tumor progression or death of any cause.

  up to 36 weeks

Secondary Outcomes (1)

• Overall Survival (OS)

  up to 36 weeks

Study Arms (1)

Leflunomide tablets (20 mg orally, once a day, before going to bed) were administered every 28 days

EXPERIMENTAL

Eligible patients will receive leflunomide tablets (20 mg orally, once a day, before bed) every 28 days until the disease progresses or adverse reactions become intolerable.

Drug: Leflunomide Pill

Interventions

Leflunomide Pill DRUG

This is a single-arm, single-center, phase II clinical study. Primarily evaluated in advanced MEN-1 neuroendocrine tumors. The initial efficacy of leflunomide tablets in second-line treatment provided evidence for phase III clinical trials.

Also known as: Leflunomide 20mg

Leflunomide tablets (20 mg orally, once a day, before going to bed) were administered every 28 days

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Cancer Institute and Hospital, Chinese Academy of Medical Sciences: lead

Study Sites (1)

National Cancer Center/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College

Beijing, 100021, China

RECRUITING

Related Publications (1)

• Ma Y, Zhu Q, Wang X, Liu M, Chen Q, Jiang L, Chi Y, Zeng YX, Zhao H, Jiao Y. Synthetic lethal screening identifies DHODH as a target for MEN1-mutated tumor cells. Cell Res. 2022 Jun;32(6):596-599. doi: 10.1038/s41422-022-00613-1. Epub 2022 Feb 15. No abstract available.

  PMID: 35169281 RESULT

MeSH Terms

Conditions

Neuroendocrine Tumors; Multiple Endocrine Neoplasia

Interventions

Leflunomide

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue; Endocrine Gland Neoplasms; Neoplasms by Site; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Isoxazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Hong Zhao

  Cancer Institute and Hospital, Chinese Academy of Medical Sciences

  STUDY DIRECTOR

Central Study Contacts

Hong Zhao

CONTACT

010-87787100; pumc95zhao@126.com

Yihebali Chi

CONTACT

01087787100

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 18, 2023
• First Posted: August 7, 2024
• Study Start: July 1, 2020
• Primary Completion: December 30, 2024
• Study Completion: December 30, 2024
• Last Updated: August 7, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)