Arumakimig (MAS825) in VEXAS syndrome: 52 weeks to see if it helps people on steroids

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a randomized, double-blind, placebo-controlled Phase 2 trial evaluating the efficacy and safety of arumakimig (MAS825) in adults with VEXAS who are on glucocorticoids. Participants are assigned 1:1 to receive arumakimig or placebo for 52 weeks, with an optional 2-year open-label extension and a subsequent safety follow-up period. The trial includes ongoing safety monitoring and a focus on long-term efficacy and tolerability in this somatic UBA1 mutation-associated autoinflammatory condition.

Why it matters

Who can join: the full rules

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.

Where it's running

• Novartis Investigative Site
  Yokohama, Kanagawa, Japan Recruiting
• Novartis Investigative Site
  Suita, Osaka, Japan Recruiting
• Novartis Investigative Site
  Hachiōji, Tokyo, Japan Recruiting
• Novartis Investigative Site
  Shinjuku-ku, Tokyo, Japan Recruiting
• Novartis Investigative Site
  Fukushima, Japan Recruiting
• Novartis Investigative Site
  Niigata, Japan Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Assessing the effectiveness of pacritinib for VEXAS syndrome

  This study is testing if the drug pacritinib can help people with VEXAS syndrome manage their symptoms better while reducing their need for steroids.

  Recruiting at 2 sites Phase 2 Swedish Orphan Biovitrum · Scottsdale, Arizona and 38 other locations · NCT06782373

• Using pacritinib to treat VEXAS syndrome

  This study is testing if the drug pacritinib can help people with VEXAS syndrome feel better and manage their symptoms.

  Recruiting Phase 1 Washington University School of Medicine · St Louis, Missouri · NCT06538181

• National registry for patients with VEXAS syndrome

  This study is collecting real-life information from adults with VEXAS syndrome to see how the disease behaves, what treatments work best, and to find important markers for diagnosis.

  Recruiting at 17 sites Technische Universität Dresden · Aachen and 16 other locations · NCT06377462

• Registry for hereditary autoinflammatory diseases

  This study is collecting information about hereditary autoinflammatory diseases to see how they affect patients of different ages and genders, with the goal of improving treatment and care for everyone affected.

  Recruiting at 112 sites University of Siena · Algiers and 111 other locations · NCT05200715

• Momelotinib treatment for VEXAS syndrome

  This trial will test whether the oral drug momelotinib helps adults with VEXAS syndrome (with or without myelodysplastic syndrome) who need daily steroids to control inflammation.

  Recruiting at 2 sites Phase 2 Groupe Francophone des Myelodysplasies · Angers and 10 other locations · NCT07098936

• Exploring genetic factors in autoimmune and autoinflammatory diseases

  This study is trying to find out how genes affect autoimmune and autoinflammatory diseases, especially VEXAS syndrome, by looking at samples from patients with known or unknown inflammatory conditions.

  Recruiting at 2 sites NYU Langone Health · New York, New York and 1 other locations · NCT06004349

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.