Advances in the Diagnosis and Management of Well-Differentiated Neuroendocrine Neoplasms.
Johannes Hofland, Gregory Kaltsas, Wouter W de Herder
https://pubmed.ncbi.nlm.nih.gov/31555796Actively Recruiting
Phase Not Applicable
Age: 18Years +
All Genders
ID06141369
Treatment of Advanced Endocrine Tumor With Individualized mRNA Neoantigen Vaccine (mRNA-0523-L001)
Led by Shanghai Jiao Tong University School of Medicine · Updated on 2026-04-09
21
Participants Needed
1
Research Sites
26 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating an individualized mRNA neoantigen vaccine (mRNA-0523-L001) for treating advanced endocrine tumors, including adrenal cortical carcinoma, medullary thyroid carcinoma, thymic neuroendocrine tumor, and pancreatic neuroendocrine tumor. These tumors often show somatic mutations and tumor-specific neoantigens, making the vaccine a promising option for patients who have failed standard treatments or have no standard options available. The study aims to assess the safety, tolerability, and preliminary efficacy of this vaccine in these challenging cancers. The vaccine is given as an intramuscular injection every 21 days for up to nine cycles. Initially, three patients will receive escalating doses to identify the maximum tolerated dose (MTD). Subsequently, 18 more patients will receive the vaccine at the MTD for all nine cycles. Treatment may continue beyond this if an objective response is observed, following specific discontinuation criteria. Participants will undergo safety and immune response monitoring at the end of each 21-day cycle, including evaluation of dose-limiting toxicity, treatment-related adverse events, and immune responses of antigen-specific T cells. Tumor response rates, disease control rates, and progression-free survival will also be assessed. The study involves regular visits and procedures to carefully track treatment effects and patient health over the course of the vaccine administration.
CONDITIONS
Brief Title
Treatment of Advanced Endocrine Tumor With Iindividualized mRNA Neoantigen Vaccine (mRNA-0523-L001)
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Subjects voluntarily sign informed consent and can comply with study visits and procedures
- Aged 18 years or older, any gender
- Confirmed advanced endocrine tumors within past 6 months, including medullary thyroid carcinoma, thymic carcinoma, adrenal cortical carcinoma, with failed or no standard treatment
- No HLA-related gene copy number variations or loss of heterozygosity detected by gene sequencing
- Advanced or metastatic lesions confirmed with sufficient frozen tissue/cells for sequencing, predicting at least one effective antigen presentation
- Expected survival of 4 months or more
- At least one measurable lesion per RECIST 1.1, untreated by recent local therapy except confirmed progression
- Eastern Cooperative Oncology Group Performance Status of 0 or 1
- No recent use of G-CSF, GM-CSF, or blood transfusions within 14 days before examination
- Agree to use reliable contraception if of childbearing potential, with negative pregnancy test for women within 7 days before first dose
- No infection with CMV, EBV, HIV, HBV, HCV, or syphilis at baseline
You will not qualify if you...
- Received chemotherapy, hormone therapy, anti-tumor treatments within 4 weeks before first dose (6 weeks for mitomycin or nitrosourea), or within 5 half-lives of immunotherapy or targeted therapy
- Major surgery other than diagnosis or biopsy within 4 weeks before first dose or planned during study
- Past allogeneic stem cell or organ transplantation, or planned organ transplant during study
- Previous tumor vaccines or cell therapies
- Uncontrolled brain or spinal cord metastases or meningitis deemed unsuitable
- Other active malignancies requiring treatment within past 2 years except certain cured cancers
- History of interstitial lung disease or pulmonary fibrosis
- Severe cardiovascular or cerebrovascular diseases including serious arrhythmias, recent acute events, or significant heart failure
- Severe or uncontrollable diseases affecting participation, including severe allergies, immunodeficiency, uncontrolled liver/kidney disease, active ulcers or infections
- Participation in other clinical trials within 4 weeks before first dose
- Current systemic corticosteroid use (except inhaled)
- Pregnant or lactating women
- Tumor invading large blood vessels with bleeding risk
- Clinically significant thyroid dysfunction unsuitable for enrollment
- Active pneumonia or uncontrolled effusions needing drainage
- Previous anti-tumor treatment side effects not resolved to mild level
- Positive hepatitis B or C tests with elevated viral load
- Investigator judgment of unsuitability for participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) for screening and enrollment
Treatment
Duration - Up to 9 cycles of 21 days each, with possible extension if response is observed
Participants receive individualized mRNA neoantigen vaccine injections every 21 days for up to 9 cycles. Treatment may continue beyond 9 cycles if an objective response is observed until discontinuation criteria are met.
Injections every 21 days for up to 9 cycles
Trial Site Locations
Total: 1 location
1
Ruijin hospital, Shanghai Jiao-Tong University School of Medicine
Shanghai, Shanghai Municipality, China, 200025
Actively Recruiting
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Research Team
L
Lei Ye
L
Luming Wu
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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