Evidence map›Paper›PMID 41753113›Full record

ArticleJournal of clinical medicine2026

PAXIS: A Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Phase 2 Study (Part 1) Followed by an Open-Label Period (Part 2) to Assess the Efficacy and Safety of Pacritinib in Patients with VEXAS Syndrome.

David B Beck, Maël Heiblig, Sinisa Savic, Marcela A Ferrada, Arsène Mekinian, Onima Chowdhury, Danielle Hammond, Lachelle D Weeks, Carmelo Gurnari, Yohei Kirino and 5 more

Registry-linked trialAbstract read
In one paragraph

Article in Journal of clinical medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06782373 (PAXIS), which is not on this map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT06782373 phase2active not recruitingnot on this map

PAXIS: A Randomized, Double-blind, Placebo-controlled Dose-finding Phase 2 Study (Part 1) Followed by an Open-label Period (Part 2) to Assess the Efficacy and Safety of Pacritinib in Patients With VEXAS Syndrome

TypeinterventionalSponsorSwedish Orphan BiovitrumRan2025 to 2028Enrolled78ConditionsVEXAS, VEXAS SyndromeArmsPacritinib, Placebo
3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. Article
  2. Review
  3. VEXAS syndrome.Nature reviews. Disease primers · 2026
    Review
  4. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

David B BeckCenter for Human Genetics and Genomics, NYU School of Medicine, New York, NY 10016, USA.ORCID 0000-0002-5884-6231
Maël HeibligHématologie Clinique, Lyon-Sud Hospital, Hospices Civils de Lyon, 69495 Pierre-Bénite, France.ORCID 0000-0003-1682-8657
Sinisa SavicLeeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Leeds LS2 9JT, UK.ORCID 0000-0001-7910-0554
Marcela A FerradaDepartment of Medicine, University of Maryland School of Medicine, Baltimore, MD 21201, USA.ORCID 0000-0003-3256-3273
Arsène MekinianSorbonne Université, Service de Médecine Interne, AP-HP, Hôpital Saint-Antoine, CEREMAIAA, 75012 Paris, France.ORCID 0000-0003-2849-3049
Onima ChowdhuryDepartment of Haematology, Oxford University Hospitals' NHS Foundation Trust, Oxford OX3 7LE, UK.ORCID 0000-0002-8150-4088
Danielle HammondDepartment of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.ORCID 0000-0002-6017-2264
Lachelle D WeeksDepartment of Medical Oncology, Division of Population Sciences, Dana Farber Cancer Institute, Boston, MA 02215, USA.ORCID 0000-0001-8726-6212
Carmelo GurnariDepartment of Biomedicine and Prevention, University of Rome Tor Vergata, 00133 Rome, Italy.ORCID 0000-0001-6829-5544
Yohei KirinoDepartment of Stem Cell and Immune Regulation, Yokohama City University Graduate School of Medicine, Yokohama 236-0004, Japan.ORCID 0000-0002-9488-661X
Sophie Georgin-LavialleSorbonne University, 75020 Paris, France.ORCID 0000-0001-6668-8854
Sarah A BuckleyMedical Affairs and Clinical Development, Sobi, Inc., Waltham, MA 02451, USA.ORCID 0000-0002-7041-8328
Raman GarchaMedical Affairs and Clinical Development, Sobi, Inc., Waltham, MA 02451, USA.
Bryan G HarderMedical Affairs and Clinical Development, Sobi, Inc., Waltham, MA 02451, USA.ORCID 0009-0003-4912-3052
Matthew J KosterDepartment of Internal Medicine, Division of Rheumatology, Mayo Clinic, Rochester, MN 55905, USA.ORCID 0000-0002-2895-6755

Funding

Sobi, Inc. Not applicable
6 · The paper itself

Abstract

VEXAS (Vacuoles, E1 ubiquitin-activating enzyme, X-linked, Autoinflammatory, Somatic) syndrome is a systemic disorder characterized by an overlap of hematologic and inflammatory features. Most patients require chronic use of moderate-to-high doses of glucocorticoids (GCs) to maintain disease control. Data on GC-sparing therapies is limited, and there have been no prospective pharmacotherapeutic trials in VEXAS syndrome published to date. Pacritinib, an oral inhibitor of IRAK1, JAK2, and ACVR1, has emerged as a promising therapeutic option for VEXAS syndrome. The PAXIS trial is the first prospective, randomized pharmacotherapeutic study conducted in this rare and severe disease. Utilizing a novel study design and disease-specific endpoints, the trial will evaluate the efficacy and safety of two dose levels of pacritinib compared with placebo in patients with VEXAS syndrome (NCT06782373, EUCTR: 2024-516347-41-00).

Indexed as

clinical trialinflammationpacritinibVEXAS

Identifiers

PMID41753113
PMCPMC12942152

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.