Actively Recruiting
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
Led by Swedish Orphan Biovitrum · Updated on 2026-04-24
78
Participants Needed
39
Research Sites
155 weeks
Total Duration
On this page
Sponsors
S
Swedish Orphan Biovitrum
Lead Sponsor
P
PSI CRO
Collaborating Sponsor
AI-Summary
What this Trial Is About
This trial is to assess the effectiveness and safety of pacritinib in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzyme, X-linked, autoinflammatory manifestations, and somatic) syndrome. 78 participants will be enrolled, randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. Randomization will be stratified by prescribed GC dose on the day of randomization.
CONDITIONS
Official Title
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Documented pathogenic UBA1 mutation at methionine-41 or nearby splice site by genetic testing
- Evidence of inflammatory involvement in at least one organ system (skin, blood vessels, joints, eyes, periorbital area, urinary tract, lungs) within 6 months prior to enrollment
- Receiving stable glucocorticoid therapy with prednisone or prednisolone between 15 and 45 mg daily for at least 4 consecutive weeks
- Karnofsky Performance Status of 50% or higher
- Adequate organ function based on liver enzymes, bilirubin, kidney function, blood counts, clotting tests, and peripheral blood blast count
- QTcF less than or equal to 450 msec in males or 470 msec in females; QRS less than or equal to 100 msec unless QTcF is less than or equal to 480 msec
- Women of child-bearing potential must have negative pregnancy tests and agree to use effective contraception
- Male participants must agree to use effective contraception
You will not qualify if you...
- Previous allogenic stem cell or solid organ transplant (except corneal)
- Using systemic glucocorticoids for conditions other than VEXAS that interfere with study
- More than one ICU admission for VEXAS flare in last 6 months
- Received 9 or more units of red blood cell transfusions in 90 days before enrollment
- Known high-risk myelodysplastic syndrome requiring treatment or transplant
- Malignancy within 1 year except certain skin cancers or carcinoma in situ
- Recent exposure to hypomethylating agents or certain anti-inflammatory or blood support therapies
- Recent use of anti-platelet therapy except low-dose aspirin
- Known multiple myeloma or certain blood protein abnormalities
- Use of strong CYP3A4 inhibitors or inducers recently
- Significant recent bleeding events
- Significant cardiovascular disease or abnormal ECG findings
- Recent arterial or venous blood clots
- Moderate or severe liver impairment or active viral hepatitis
- Uncontrolled HIV or detectable viral load on antiretrovirals
- Positive tuberculosis test
- History of disseminated mycobacterial infection
- Concurrent participation in other interventional trials or recent experimental therapy
- Pregnant, planning pregnancy, or breastfeeding
- Active infection requiring systemic antibiotics except prophylactic or chronic use
- Allergy to pacritinib or its inactive ingredients including microcrystalline cellulose, polyethylene glycol, magnesium stearate
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 39 locations
1
Mayo Clinic - Scottsdale
Scottsdale, Arizona, United States, 85259
Actively Recruiting
2
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Actively Recruiting
3
Mayo Clinic - Rochester
Rochester, Minnesota, United States, 55905
Actively Recruiting
4
NYU Langone Health
New York, New York, United States, 10016
Actively Recruiting
5
Cleveland Clinic - Cleveland
Cleveland, Ohio, United States, 44195
Actively Recruiting
6
The James Cancer Hospital and Solove Research Institute
Columbus, Ohio, United States, 43210
Actively Recruiting
7
UT MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
8
University of Utah Healthcare
Salt Lake City, Utah, United States, 84132
Actively Recruiting
9
Fred Hutchinson Cancer Center
Seattle, Washington, United States, 98109
Actively Recruiting
10
Vancouver Coastal Health Research Institute
Vancouver, British Columbia, Canada, V5Z 1M9
Actively Recruiting
11
Queen Elizabeth II Health Sciences Center
Halifax, Nova Scotia, Canada, B3H 2Y9
Actively Recruiting
12
Princess Margaret Cancer Centre
Toronto, Ontario, Canada, M5G 2M9
Actively Recruiting
13
Hospital du Sacre-Coeur in Montreal
Montreal, Quebec, Canada, H4J 1C5
Actively Recruiting
14
Lille University Hospital Center
Lille, France, 59037
Actively Recruiting
15
Saint-Antoine Hospital - APHP
Paris, France, 75012
Actively Recruiting
16
Tenon Hospital - APHP
Paris, France, 75020
Actively Recruiting
17
Hospices Civils de Lyon - Lyon Sud
Pierre-Bénite, France, 69310
Actively Recruiting
18
University Hospital Center of Poitiers
Poitiers, France, 86000
Actively Recruiting
19
IUCT-Oncopole
Toulouse, France, 31100
Actively Recruiting
20
University Hospital Tuebingen, Medical Clinic II, Hematology, Oncology, Clinical Immunology and Rheumatology
Tübingen, Baden-Wurttemberg, Germany, 72076
Actively Recruiting
21
Hospital Rechts der Isar of the Technical University of Munich, Clinic and Polyclinic for Internal Medicine III: Hematology and Internal Oncology
Munich, Bavaria, Germany, 81675
Actively Recruiting
22
University Hospital Hamburg-Eppendorf
Hamburg, Free and Hanseatic City of Hamburg, Germany, 20246
Actively Recruiting
23
University Hospital Duesseldorf
Düsseldorf, North Rhine-Westphalia, Germany, 40225
Actively Recruiting
24
University Hospital Carl Gustav Carus Dresden, Medical Clinic and Polyclinic I
Dresden, Saxony, Germany, 01307
Actively Recruiting
25
University Hospital Schleswig-Holstein
Lübeck, Schleswig-Holstein, Germany, 23538
Actively Recruiting
26
Hospital San Raffaele, IRCCS, Unit of Immunology, Rheumatology, Allergy and Rare Diseases
Milan, Italy, 20132
Actively Recruiting
27
University Hospital of Padova, Rheumatology Unit, Department of Medicine - DIMED
Padova, Italy, 35128
Actively Recruiting
28
AUSL of Reggio Emilia - Hospital Arcispedale S. Maria Nuova, Complex Structure of Rheumatology
Reggio Emilia, Italy, 42123
Actively Recruiting
29
Foundation PTV - Polyclinic Tor Vergata Biomedicine and prevention
Roma, Italy, 00133
Actively Recruiting
30
Fukushima Medical University Hospital
Fukushima, Japan, 960-1295
Actively Recruiting
31
Nagasaki University Hospital
Nagasaki, Japan, 852-8501
Actively Recruiting
32
Yokohama City University Hospital
Yokohama, Japan, 236-0004
Actively Recruiting
33
Hospital Clinic of Barcelona
Barcelona, Spain, 08036
Actively Recruiting
34
Catalan Institute of Oncology, Hospital Duran i Reynals, Department of Clinical Hematology
L'Hospitalet de Llobregat, Spain, 08908
Actively Recruiting
35
University Clinical Hospital of Salamanca
Salamanca, Spain, 37007
Actively Recruiting
36
St James's University Hospital
Leeds, United Kingdom, LS9 7TF
Actively Recruiting
37
Royal Free Hospital
London, United Kingdom, NW3 2QG
Actively Recruiting
38
King's College Hospital, Department of Hematology
London, United Kingdom, SE5 9RS
Actively Recruiting
39
Churchill Hospital
Oxford, United Kingdom, OX3 7LE
Actively Recruiting
Research Team
S
Study Physician
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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