Old arthritis drug tested against rare tumor syndrome – but trial stalls
NCT ID NCT05605587
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested leflunomide, a drug used for rheumatoid arthritis, in 3 adults with MEN1 syndrome—a genetic condition that raises the risk of multiple tumors. The goal was to see if 6 months of daily leflunomide could shrink or stabilize tumors. However, the trial was terminated early, so we have very little information on whether it worked.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Leflunomide (20 mg daily for 6 months)
- What this could lead to
- If it works, leflunomide could offer a new way to slow or shrink tumors in people with MEN1 syndrome.
- What could go wrong
- This was a very small, early trial that ended early, so we don't have enough data to know if it works. Leflunomide can also cause side effects like liver problems or low blood counts.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Not a phased trial
Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.
- Participants
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3 people
The number who actually took part.
- Started
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May 2023
- Finished
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Jan 2026
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Adult (≥18 years) patients with known pathogenic or likely pathogenic MEN1-germline mutation and at least 1 associated tumor lesion OR hormonal syndrome Exclusion Criteria: * uncontrolled arterial hypertension, defined as blood pressure \>160/100 mmHg * Impaired kidney function, defined as creatinine clearance \<50ml/min * Impaired liver function, defined as bilirubin or liver transaminases \>3 times upper normal range * Cytopenia, defined as one or several of the following: hemogloin \<100 g/l, leucopenia \<2x109/l, thrombocytopenia \<100x109/l
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Unispital Basel
Basel, 4053, Switzerland