VEXAS syndrome

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ORPHA:596753OMIM:301054M35.8
Who is this for?
Show terms as
5Active trials7Specialists8Treatment centers

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

5 events
Sep 2025Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study

National Heart, Lung, and Blood Institute (NHLBI) — NA

TrialENROLLING BY INVITATION
May 2025A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)

Swedish Orphan Biovitrum — PHASE2

TrialRECRUITING
Feb 2025Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome

Washington University School of Medicine — PHASE1

TrialRECRUITING
Nov 2024HRQoL and Financial Toxicity in Patients With VEXAS Syndrome

Gruppo Italiano Malattie EMatologiche dell'Adulto

TrialRECRUITING
Mar 2024Multicenter, Interdisciplinary National VEXAS Registry With Accompanying Biomaterial Collection

Technische Universität Dresden

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for VEXAS syndrome.

5 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

5 recruitingView all trials with filters →
Phase 21 trial
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
Phase 2
Actively Recruiting
PI: Study Physician (Sobi, Inc.) · Sites: Scottsdale, Arizona; Baltimore, Maryland +18 more · Age: 1899 yrs
Phase 11 trial
Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome
Phase 1
Actively Recruiting
PI: Meagan A Jacoby, M.D., Ph.D. (Washington University School of Medicine) · Sites: St Louis, Missouri · Age: 1899 yrs
N/A1 trial
Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study
N/A
Enrolling by Invitation
PI: Bhavisha A Patel, M.D. (National Heart, Lung, and Blood Institute (NHLBI)) · Sites: Bethesda, Maryland · Age: 2120 yrs
Other2 trials
HRQoL and Financial Toxicity in Patients With VEXAS Syndrome
Actively Recruiting
· Sites: Ancona; Bergamo +15 more · Age: 1899 yrs
Multicenter, Interdisciplinary National VEXAS Registry With Accompanying Biomaterial Collection
Actively Recruiting
PI: Katja Sockel, Dr. med. (TU Dresden) · Sites: Aachen; Chemnitz +11 more · Age: 1899 yrs

Specialists

7 foundView all specialists →
MM
Maël HEIBLIG, MD
Specialist
PI on 1 active trial
MP
Meagan A Jacoby, M.D., Ph.D.
St Louis, Missouri
Specialist

Rare Disease Specialist

PI on 1 active trial
BM
Bhavisha A Patel, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 4 active trials
DP
David Beck, MD, PhD
Specialist
PI on 1 active trial
KM
Katja Sockel, Dr. med.
Specialist
PI on 1 active trial
AM
Arsene MEKINIAN
Specialist
PI on 1 active trial

Treatment Centers

8 centers
⚗️ Trial Site

National Institutes of Health Clinical Center

📍 Bethesda, Maryland

👤 Payal P Khincha, M.D.

👤 Christopher Grunseich, M.D.

⚗️ Trial Site

Washington University School of Medicine

📍 St Louis, Missouri

👤 Meagan A Jacoby, M.D., Ph.D.

👤 Janssen Research & Development, LLC Clinical Trial

⚗️ Trial Site

Dana Farber Cancer Institute

📍 Boston, Massachusetts

👤 Janssen Research & Development, LLC Clinical Trial

⚗️ Trial Site

Fred Hutchinson Cancer Center

📍 Seattle, Washington

👤 Sawa M Ito, MD, PhD

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to VEXAS syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about VEXAS syndrome

Disease timeline:

New recruiting trial: HRQoL and Financial Toxicity in Patients With VEXAS Syndrome

A new clinical trial is recruiting patients for VEXAS syndrome

New recruiting trial: Multicenter, Interdisciplinary National VEXAS Registry With Accompanying Biomaterial Collection

A new clinical trial is recruiting patients for VEXAS syndrome

New trial: Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study

Phase NA trial recruiting.

New trial: Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) S

Phase PHASE1 trial recruiting. Pacritinib

New trial: A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)

Phase PHASE2 trial recruiting. Pacritinib

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about VEXAS syndrome

Are there clinical trials for VEXAS syndrome?

Yes — 5 recruiting clinical trials are currently listed for VEXAS syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat VEXAS syndrome?

7 specialists and care centers treating VEXAS syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.