Can a new drug tame VEXAS syndrome and cut steroid dependence?
NCT ID NCT07748624
First seen Aug 06, 2026 · Last updated Aug 07, 2026 · Updated 1 time
Summary
This phase 2 trial is testing whether an investigational drug called arumakimig can improve symptoms of VEXAS syndrome, a rare genetic autoinflammatory condition. The study will compare arumakimig against a placebo in about 120 adults with VEXAS, with the goal of seeing if the drug can reduce disease activity and allow patients to lower their steroid use. After the initial 52-week period, participants may continue into an open-label extension to assess long-term safety and effectiveness.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Arumakimig (MAS825), an investigational drug, given as an injection
- What this could lead to
- If successful, arumakimig could offer a new treatment option for VEXAS syndrome, potentially reducing symptoms and the need for steroids.
- What could go wrong
- This is an early-phase trial, so the drug may not work as hoped or may cause side effects. Results from this study will not be conclusive.
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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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 120 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Oct 2026
An estimate. Start dates often move.
- Expected to finish
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Apr 2031
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
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.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Male and female participants aged ≥18 years at screening. * Somatic mutation in UBA1 gene known to be associated with VEXAS. * Participants must have at least two manifestations of VEXAS at screening or in the past 6 months. * Participants must be able to start treatment for Pneumocystis jiroveci pneumonia (PJP) during the study if indicated according to the local guidelines. * Ability to communicate well with the Investigator, understand and agree to comply with the requirements of the study. Exclusion Criteria: * Participants meeting any of the following criteria are not eligible for this study: * Positive serology for hepatitis B surface antigen (HBsAg) excludes the participant. HBsAg negative participants who are hepatitis B core antibody (HBcAb) positive are also excluded unless protocol-defined criteria are met. * Participants with a positive HCV antibody test should have HCV ribonucleic acid (RNA) levels measured. Participants with positive (detectable) HCV RNA must be excluded. Chronic hepatitis C patients who have completed HCV anti-viral treatment must be HCV-RNA negative at least 12 weeks after treatment before randomization to be eligible. * Active viral, bacterial, or other infections requiring systemic treatment at the time of screening or randomization, or history of recurrent clinically significant infection or of recurrent bacterial infections. * Known or suspected Human Immunodeficiency Virus (HIV) infection. Should it be required by local regulations and/or considered appropriate by the Investigator, an HIV test can be performed locally to confirm eligibility. * Live vaccinations within a certain period prior to arumakimig treatment. Live vaccines are prohibited during the trial and up to a certain period following the last dose of arumakimig. * History of malignancy of any organ system, including post-transplant lymphoproliferative disorder (except for skin Bowen's disease, completely treated and resolved, localized squamous or basal cell carcinoma of the skin or actinic keratosis that have been treated with no evidence of recurrence in the past 12 weeks, in situ cervical cancer or non-invasive malignant colon polyps that have been removed), treated or untreated, within a protocol-defined period, regardless of whether there is evidence of local recurrence or metastases. * History of or current hepatic disease (moderate to severe Hepatic Impairment as per Child-Pugh classification), including but not limited to, acute or chronic hepatitis (for Hepatitis B or C), cirrhosis or hepatic failure. * Participants of child-bearing potential who do not agree to comply with required contraceptive use as outlined in the protocol. Other protocol-defined inclusion/exclusion criteria may apply.
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Experimental drug pacritinib takes aim at rare inflammatory VEXAS syndrome
- Hope for rare disease: new drug trial for VEXAS syndrome begins
- Massive study to uncover hidden link between blood mutations and immune disorders
- New study uses DNA tests to catch blood cancers before they start
- Could a stem cell transplant tame VEXAS syndrome?