Neuroendocrine TumorsPhase 2NCT06540937
Phase II Clinical Study of Leflunomide in the Treatment of MEN-1 Neuroendocrine Tumor
A Phase 2 study testing Leflunomide Pill in people with Neuroendocrine Tumors, run by Cancer Institute and Hospital, Chinese Academy of Medical Sciences at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Neuroendocrine Tumors
- What is being tested?
- Leflunomide Pill
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2020to 30 Dec 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
Yihebali Chi contact
China1 site, 1 recruiting
- Recruiting
National Cancer Center/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Beijing, 100021
Hong Zhao, 010-87787100, pumc95zhao@126.com
Yihebali Chi, 010-87787100
About this study
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.
Read the detailed description
- Treatment methods 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.
- Follow-up Eligible patients should follow the requirements of the study protocol and continue to the end of the study. The purpose of the follow-up is to know whether the subjects have adverse reactions to the treatment effect and take appropriate treatment. Evaluation should be conducted every two cycles. If the test is terminated before disease progression, the subjects should be followed up every 6 weeks for imaging examination to observe whether the tumor progresses. Other antitumor therapy, hepatotoxic drugs (phenylbutazone, phenytoin, methotrexate, methyldopa, rifampicin) and traditional Chinese medicine were not allowed before the group withdrawal. At each follow-up, the patient's history is taken, and the following examinations are performed: physical examination; Blood routine, urine routine, liver and kidney function, serum tumor markers, electrocardiogram; Tumor imaging (e.g., chest and abdominal CT); Blood biomarker test; other tests required for the condition.
- Tumor tissue/blood biomarker examination In this project, the mutation of MEN-1 gene of the patient should be tested, and blood samples of the patient and tumor tissue samples remaining during routine diagnosis and treatment should be collected. The remaining samples will be returned to the Cancer Hospital of Chinese Academy of Medical Sciences for storage. The results of these tests will determine whether the patient can participate in the study.
- Conditions
- Neuroendocrine Tumors
- Being tested
- Leflunomide Pill
- Keywords
- Multiple Endocrine Neoplasia, Leflunomide
- Medical subject headings
- Neuroendocrine Tumors, Multiple Endocrine Neoplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Leflunomide PillDrug
Also called Leflunomide 20mg
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Leflunomide Pill |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: up to 36 weeks
Secondary outcomes (1)
Overall Survival (OS)
OS is defined as the time from the date of randomization until death of any cause.
Measured: up to 36 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2024
- Study start1 Jul 2020
- Primary completion30 Dec 2024 (estimated)
- Study completion30 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Neuroendocrine TumorsStudies of the same condition, open and under way.
- More trials run by Cancer Institute and Hospital, Chinese Academy of Medical SciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Hong Zhao, Study director, Cancer Institute and Hospital, Chinese Academy of Medical Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06540937
- Sponsor's protocol ID
- NCC2409
- Status last verified
- Aug 2024
- First submitted
- 18 Feb 2023
- Last updated
- 7 Aug 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 35169281
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Clinical Study of Leflunomide in the Treatment of MEN-1 Neuroendocrine Tumor
- ClinicalTrials.gov ID
- NCT06540937
- Acronym / sponsor ID
- NCC2409
- Conditions
- Neuroendocrine Tumors
- Keywords
- Multiple Endocrine Neoplasia, Leflunomide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical SciencesUniversity, hospital or other
- Study start
- 1 Jul 2020 (actual)
- Primary completion
- 30 Dec 2024 (estimated)
- Study completion
- 30 Dec 2024 (estimated)
- First posted
- 7 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
- Treatment methods 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.
- Follow-up Eligible patients should follow the requirements of the study protocol and continue to the end of the study. The purpose of the follow-up is to know whether the subjects have adverse reactions to the treatment effect and take appropriate treatment. Evaluation should be conducted every two cycles. If the test is terminated before disease progression, the subjects should be followed up every 6 weeks for imaging examination to observe whether the tumor progresses. Other antitumor therapy, hepatotoxic drugs (phenylbutazone, phenytoin, methotrexate, methyldopa, rifampicin) and traditional Chinese medicine were not allowed before the group withdrawal. At each follow-up, the patient's history is taken, and the following examinations are performed: physical examination; Blood routine, urine routine, liver and kidney function, serum tumor markers, electrocardiogram; Tumor imaging (e.g., chest and abdominal CT); Blood biomarker test; other tests required for the condition.
- Tumor tissue/blood biomarker examination In this project, the mutation of MEN-1 gene of the patient should be tested, and blood samples of the patient and tumor tissue samples remaining during routine diagnosis and treatment should be collected. The remaining samples will be returned to the Cancer Hospital of Chinese Academy of Medical Sciences for storage. The results of these tests will determine whether the patient can participate in the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Leflunomide tablets (20 mg orally, once a day, before going to bed) were administered every 28 days | Experimental | Drug: Leflunomide PillEligible 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. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Leflunomide PillAlso: Leflunomide 20mgThis 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. | Drug | Leflunomide tablets (20 mg orally, once a day, before going to bed) were administered every 28 days |
Primary outcome
Overall response rate (ORR) assessed by RECIST 1.1
Time frame: up to 36 weeks
ORR is defined as the percentage of patients with a complete response (CR) or partial response (PR) according to RECIST v1.1.
Progression-free survival (PFS)
Time frame: up to 36 weeks
PFS is defined as the duration from the date of randomization to the onset of tumor progression or death of any cause.
1 secondary outcome
Overall Survival (OS)
Time frame: up to 36 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2024.
- Results publication: PubMed 35169281Ma 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yihebali Chi contact
Hong Zhao study director
Cancer Institute and Hospital, Chinese Academy of Medical Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Leflunomide Pill in people with Neuroendocrine Tumors, run by Cancer Institute and Hospital, Chinese Academy of Medical Sciences at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 7 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Hong Zhao (010-87787100, pumc95zhao@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (ORR) assessed by RECIST 1.1 (time frame: up to 36 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06540937, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2024.