A 52-Week Study Evaluating the Efficacy and Safety of Arumakimig (MAS825) in Participants With VEXAS Followed by Open-Label Extension (OLE) Period

NCT07748624 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: CMAS825G122032026-527354-39
• Study Type: interventional
• Target: 120
• Locations: 0 countries
• Sites: N/A

Brief 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. In addition, the study will evaluate the long-term efficacy, safety and tolerability of arumakimig in this population.

Conditions

VEXAS Syndrome

Keywords

VEXAS syndrome; MAS825; Arumakimig; somatic mutation; ubiquitin-like modifier activating enzyme (UBA1); glucocorticoid taper; vacuoles; E1 Enzyme; Autoinflammatory; Arthritis; arthralgia

Outcome Measures

Primary Outcomes (1)

• Number of participants achieving improvement of key VEXAS manifestations and oral glucocorticoid (GC) reduction at 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.

  From baseline up to Week 52

Secondary Outcomes (9)

• Number of participants achieving Overall Clinical Response (OCR) at Week 52

  From baseline up to Week 52

• Number of participants achieving resolution of VEXAS manifestations

  From baseline up to Week 52

• Number of participants with oral glucocorticoid reduction

  From baseline up to Week 52

• Total number of flare-free days over 52 weeks

  Up to 52 weeks

• Number of participants achieving Hematologic Improvement - Erythroid (HI-E) during the double-blind treatment period

  From baseline up to Week 52

Study Arms (2)

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.

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.

Drug: Arumakimig; Drug: Placebo; Drug: Oral glucocorticoids

Interventions

Arumakimig DRUG

Arumakimig Injection

Also known as: MAS825

Arumakimig; Placebo

Placebo DRUG

Placebo Injection

Placebo

Oral glucocorticoids DRUG

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.

Arumakimig; Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

MeSH Terms

Conditions

VEXAS syndrome; Arthritis; Arthralgia

Interventions

Glucocorticoids

Condition Hierarchy (Ancestors)

Joint Diseases; Musculoskeletal Diseases; Pain; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Physiological Effects of Drugs; Pharmacologic Actions; Chemical Actions and Uses

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 31, 2026
• First Posted: August 6, 2026
• Study Start (Estimated): October 30, 2026
• Primary Completion (Estimated): December 26, 2028
• Study Completion (Estimated): April 21, 2031
• Last Updated: August 6, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will share