ClinicalTrials.gov record NCT07748624
A 52-Week Study Evaluating the Efficacy and Safety of Arumakimig (MAS825) in Participants With VEXAS Followed by Open-Label Extension (OLE) Period
VEX-AR: Randomized, Double-Blind, Placebo-Controlled, 52-Week Phase 2 Study Evaluating the Efficacy and Safety of Arumakimig (MAS825) in Participants With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome, Followed by an Open-Label Extension Period
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This randomized, double-blind, placebo-controlled Phase 2 study evaluates arumakimig (MAS825) in people with VEXAS who are receiving glucocorticoids. The main blinded treatment period lasts 52 weeks, with participants randomized 1:1 to arumakimig or placebo while continuing background oral glucocorticoids; glucocorticoid tapering may begin after the first 2 weeks per investigator judgement. After Week 52, participants may enter a 104-week open-label extension (continuing to Week 156). A 16-week safety follow-up is required after the double-blind period or after the open-label extension.
What it is studying. The study tests arumakimig (MAS825) compared with placebo to assess effects on key VEXAS clinical manifestations and protocol-defined reduction of oral glucocorticoids through 52 weeks, and it evaluates longer-term safety and tolerability during an optional 104-week open-label extension.
What the researchers hope to learn. Scientifically, the study aims to measure: the number of participants who achieve improvement of key VEXAS manifestations together with protocol-defined oral glucocorticoid reduction at Week 52 (primary outcome); overall clinical response, resolution of VEXAS manifestations, glucocorticoid reduction, total flare-free days, hematologic improvements (erythroid and platelets), participant-reported disease worsening, time to death during the double-blind period, and the number and nature of adverse events and serious adverse events (safety) through the follow-up period.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The purpose of this study is to evaluate clinical efficacy and safety of arumakimig (MAS825) compared to placebo in patients with Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) syndrome. In addition, the study will evaluate the long-term efficacy, safety and tolerability of arumakimig in this population.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Arumakimig
- Placebo
- Oral glucocorticoids
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
This is a randomized, double-blind, placebo-controlled study with a 52-week duration evaluating the efficacy and safety of arumakimig in participants with VEXAS who are receiving glucocorticoids. Participants will be randomized 1:1 to either arumakimig or placebo. Following the double-blind period, participants may have the option to enter a 2-year (104-week) open-label extension (OLE) period, continuing until Week 156. A 16-week safety follow-up period must be completed after the OLE (up to Week 172) or after the double-blind period (up to Week 68).
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants are randomized to receive either arumakimig injection or a placebo injection during a 52-week double-blind period while continuing oral glucocorticoid treatment. After the first 2 weeks, glucocorticoid tapering may begin depending on disease status and the Investigator's judgement, and participants who complete the double-blind period may enter a 104-week (2-year) open-label extension to receive arumakimig through Week 156. Study activities include regular safety monitoring (vital signs, electrocardiograms, laboratory tests and reporting of adverse events), assessments of symptoms, and blood measures such as C-reactive protein, hemoglobin and platelets; a 16-week safety follow-up is required after the double-blind period or after the extension. If indicated by local guidelines, participants must be able to start treatment for Pneumocystis jiroveci pneumonia (PJP) during the study.
How the study is structured. Participants are randomly assigned 1:1 to receive either arumakimig (MAS825) or placebo in a double-blind, parallel-group format for 52 weeks. All participants continue background oral glucocorticoids; tapering of glucocorticoids may start after the first two weeks depending on disease status and investigator judgement. After the blinded 52-week period, eligible participants may join a 104-week open-label extension to receive arumakimig through Week 156. A 16-week safety follow-up must be completed after the blinded period or after the open-label extension.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
- The listed sites are in Japan. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on VEXAS syndrome
Important dates
- Study start
- Oct 30, 2026 (estimated)
- Primary completion
- Dec 26, 2028 (estimated)
- Study completion
- Apr 15, 2031 (estimated)
- Target enrollment
- 120 participants (estimated)
- Last updated on registry
- Sep 29, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Novartis Pharmaceuticals
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Novartis Pharmaceuticals 📞 1-888-669-6682 ✉ novartis.email@novartis.com
- Novartis Pharmaceuticals 📞 +41613241111
Participating sites (3)
- Novartis Investigative SiteRecruiting
Yokohama, Kanagawa, 2360004 · Japan
- Novartis Investigative SiteRecruiting
Suita, Osaka, 5650871 · Japan
- Novartis Investigative SiteRecruiting
Hachiōji, Tokyo, 1930998 · Japan
Terms used on this page · explanations by MedicalUpdates.ai
- placebo
- A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
- at screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- randomization
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- double-blind
- Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
- placebo-controlled
- A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
- glucocorticoids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- 1:1
- How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
- open-label extension
- A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.
- safety follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this website; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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