VEXAS SyndromePhase 2NCT07748624
A 52-Week Study Evaluating the Efficacy and Safety of Arumakimig (MAS825) in Participants With VEXAS Followed by Open-Label Extension (OLE) Period
A Phase 2 study testing Arumakimig and Oral glucocorticoids in people with VEXAS Syndrome, run by Novartis Pharmaceuticals at 4 sites in Japan.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- VEXAS Syndrome
- What is being tested?
- Arumakimig and Oral glucocorticoids
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 120 participants
- Where
- 4 sitesJapan
- Study dates
- 30 Oct 2026to 15 Apr 2031
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
Japan4 sites, 4 recruiting
- Recruiting
Novartis Investigative Site
Fukushima, 9601295
- Recruiting
Novartis Investigative Site
Hachiōji, Tokyo, 1930998
- Recruiting
Novartis Investigative Site
Suita, Osaka, 5650871
- Recruiting
Novartis Investigative Site
Yokohama, Kanagawa, 2360004
About this study
Official title: 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
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.
Read the detailed description
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).
- Conditions
- VEXAS Syndrome
- Being tested
- Arumakimig, Placebo, Oral glucocorticoids
- Keywords
- VEXAS syndrome, MAS825, Arumakimig, somatic mutation, ubiquitin-like modifier activating enzyme (UBA1), glucocorticoid taper, vacuoles, E1 Enzyme, Autoinflammatory, Arthritis, arthralgia
- Medical subject headings
- VEXAS syndrome, Arthritis, Arthralgia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ArumakimigDrug
Also called MAS825
Arumakimig Injection
PlaceboDrug
Placebo Injection
Oral glucocorticoidsDrug
Background therapy with glucocorticoids. After the first 2 weeks of the study, participants may begin with glucocorticoid tapering depending on the disease status and the Investigator's judgement.
| Study group | Role | What they receive |
|---|---|---|
| Arumakimig | Experimental | Arumakimig from Day 1 to Week 52 during the double-blind period. Eligible participants may then continue into an open-label extension and receive arumakimig through Week 156. Receives: Drug: Arumakimig; Drug: Oral glucocorticoids |
| Placebo | Placebo comparator | Placebo from Day 1 to Week 52 during the double-blind period. Eligible participants may then continue into an open-label extension and receive arumakimig through Week 156. Receives: Drug: Arumakimig; Drug: Placebo; Drug: Oral glucocorticoids |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants achieving improvement of key VEXAS manifestations and oral glucocorticoid (GC) reduction at Week 52
Response is defined as meeting both criteria:
- Clinical domain: In participants with clinical disease activity in any domain at baseline, complete resolution of clinical manifestations related to active disease in at least one affected domain. In participants with no clinical disease activity in any domain at baseline, continued absence of clinical manifestations in all domains at Week 52.
AND B) Protocol-defined glucocorticoid reduction through 52 weeks.
Measured: From baseline up to Week 52
Secondary outcomes (9)
Number of participants achieving Overall Clinical Response (OCR) at Week 52
Achieving OCR at Week 52 is defined as meeting all the following criteria:
- Absence of active inflammatory manifestations of VEXAS
- Protocol-defined reduction in oral glucocorticoid.
- Protocol-defined reduction in C reactive protein.
Measured: From baseline up to Week 52
Number of participants achieving resolution of VEXAS manifestations
In participants with clinical disease activity in any domain at baseline, achieving complete resolution of clinical manifestations.
Measured: From baseline up to Week 52
Number of participants with oral glucocorticoid reduction
Number of participants achieving protocol-defined glucocorticoid dose reduction through 52 weeks.
Measured: From baseline up to Week 52
Total number of flare-free days over 52 weeks
Total cumulative number of days over 52 weeks meeting the protocol-defined criteria for a flare-free day.
Measured: Up to 52 weeks
Number of participants achieving Hematologic Improvement - Erythroid (HI-E) during the double-blind treatment period
Hematologic Improvement - Erythroid (HI-E) during the double-blind 52-week treatment period among participants with baseline hemoglobin within the protocol-defined range, according to modified International Working Group (IWG) and protocol-defined response criteria.
Measured: From baseline up to Week 52
Number of participants achieving Hematologic Improvement - Platelets (HI-P) during the double-blind treatment period
Hematologic Improvement - Platelets (HI-P) during the double-blind 52-week treatment period among participants with baseline platelets within the protocol-defined range, according to modified International Working Group (IWG) and protocol-defined response criteria.
Measured: From baseline up to Week 52
Number of participants without worsening disease according to a participant-reported questionnaire
Participants without protocol-defined worsening of disease according to a participant-reported questionnaire.
Measured: From baseline up to Week 52
Time to death during the double-blind treatment period
Assessment of mortality through Week 52 in each treatment arm.
Measured: Up to 52 weeks
Number of participants with adverse events (AEs) and serious adverse events (SAEs)
Number of participants with AEs and SAEs, including changes in vital signs, electrocardiograms and laboratory values qualifying and reported as AEs.
Measured: Up to 172 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2026
- Study start30 Oct 2026 (estimated)
- Primary completion26 Dec 2028 (estimated)
- Study completion15 Apr 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for VEXAS SyndromeStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07748624
- Sponsor's protocol ID
- CMAS825G12203
- Other IDs
- 2026-527354-39
- Status last verified
- Oct 2026
- First submitted
- 31 Jul 2026
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07748624
- Acronym / sponsor ID
- CMAS825G12203
- Conditions
- VEXAS Syndrome
- Keywords
- VEXAS syndrome, MAS825, Arumakimig, somatic mutation, ubiquitin-like modifier activating enzyme (UBA1), glucocorticoid taper, vacuoles, E1 Enzyme
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 30 Oct 2026 (estimated)
- Primary completion
- 26 Dec 2028 (estimated)
- Study completion
- 15 Apr 2031 (estimated)
- First posted
- 6 Aug 2026 (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.
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.
Detailed description
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arumakimig | Experimental | Drug: Arumakimig, Drug: Oral glucocorticoidsArumakimig from Day 1 to Week 52 during the double-blind period. Eligible participants may then continue into an open-label extension and receive arumakimig through Week 156. |
| Placebo | Placebo comparator | Drug: Arumakimig, Drug: Placebo, Drug: Oral glucocorticoidsPlacebo from Day 1 to Week 52 during the double-blind period. Eligible participants may then continue into an open-label extension and receive arumakimig through Week 156. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
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 |
|---|---|---|
| ArumakimigAlso: MAS825Arumakimig Injection | Drug | Arumakimig, Placebo |
| PlaceboPlacebo Injection | Drug | Placebo |
| Oral glucocorticoidsBackground therapy with glucocorticoids. After the first 2 weeks of the study, participants may begin with glucocorticoid tapering depending on the disease status and the Investigator's judgement. | Drug | Arumakimig, Placebo |
Primary outcome
Number of participants achieving improvement of key VEXAS manifestations and oral glucocorticoid (GC) reduction at Week 52
Time frame: From baseline up to Week 52
Response is defined as meeting both criteria: A) Clinical domain: In participants with clinical disease activity in any domain at baseline, complete resolution of clinical manifestations related to active disease in at least one affected domain. In participants with no clinical disease activity in any domain at baseline, continued absence of clinical manifestations in all domains at Week 52. AND B) Protocol-defined glucocorticoid reduction through 52 weeks.
9 secondary outcomes
Number of participants achieving Overall Clinical Response (OCR) at Week 52
Time frame: From baseline up to Week 52
Number of participants achieving resolution of VEXAS manifestations
Time frame: From baseline up to Week 52
Number of participants with oral glucocorticoid reduction
Time frame: From baseline up to Week 52
Total number of flare-free days over 52 weeks
Time frame: Up to 52 weeks
Number of participants achieving Hematologic Improvement - Erythroid (HI-E) during the double-blind treatment period
Time frame: From baseline up to Week 52
Number of participants achieving Hematologic Improvement - Platelets (HI-P) during the double-blind treatment period
Time frame: From baseline up to Week 52
Number of participants without worsening disease according to a participant-reported questionnaire
Time frame: From baseline up to Week 52
Time to death during the double-blind treatment period
Time frame: Up to 52 weeks
Number of participants with adverse events (AEs) and serious adverse events (SAEs)
Time frame: Up to 172 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2026.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.
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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.
Novartis Pharmaceuticals contact
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Arumakimig and Oral glucocorticoids in people with VEXAS Syndrome, run by Novartis Pharmaceuticals at 4 sites in Japan.
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 5 inclusion and 11 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: looking for participants now, as of the sponsor's last update on 13 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Japan. 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. Some participants in this study may receive a placebo. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (1-888-669-6682, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants achieving improvement of key VEXAS manifestations and oral glucocorticoid (GC) reduction at Week 52 (time frame: From baseline up to Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 13 Oct 2026.