Actively Recruiting

Phase 2
Age: 18Years +
All Genders
ID06782373

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

Led by Swedish Orphan Biovitrum · Updated on 2026-04-24

78

Participants Needed

39

Research Sites

72 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

Researchers are evaluating the effectiveness and safety of pacritinib in people with VEXAS syndrome, a rare inflammatory condition caused by specific genetic mutations. This randomized, placebo-controlled phase 2 trial aims to prevent flares of VEXAS symptoms after reducing glucocorticoid (GC) treatment. The study will include adults aged 18 and older who have been on stable GC therapy for at least four weeks and have inflammatory involvement of various organs linked to VEXAS syndrome. Participants will be randomly assigned to one of three groups: pacritinib dose A, pacritinib dose B plus placebo, or placebo alone, all taken orally for up to 24 weeks without knowing which they receive. After this double-blind period, all participants will have the option to receive open-label pacritinib for up to 48 weeks. Those benefiting from pacritinib may continue treatment for an additional year in an extension period. If a participant stops treatment, they will be followed for 30 days after. Throughout the study, participants will have regular assessments to monitor clinical response, flare frequency, blood parameters, and quality of life using patient-reported questionnaires. Blood tests will measure drug levels and inflammation markers like C-reactive protein, erythrocyte sedimentation rate, and ferritin. The main outcome is overall clinical response by week 24. Safety and efficacy data will be collected until about two years after the first dose of the last participant.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Adults aged 18 years or older
  • Confirmed UBA1 gene mutation at methionine-41 or nearby splice site by specific genetic testing
  • Evidence of inflammatory involvement in at least one organ system related to VEXAS within 6 months before enrollment
  • Currently receiving stable glucocorticoid therapy with prednisone or prednisolone between 15 and 45 mg daily
  • Karnofsky Performance Status of 50% or higher
  • Adequate organ function based on liver enzymes, bilirubin, kidney function, neutrophil count, coagulation tests, platelet count, and peripheral blast count
  • QTcF interval within specified limits by sex and heart rhythm criteria
  • Women of child-bearing potential must have negative pregnancy tests before enrollment
  • Agreement to use effective contraception during and for 30 days after trial therapy
Not Eligible

You will not qualify if you...

  • Prior allogenic hematopoietic stem cell or solid organ transplant (except corneal)
  • Use of systemic glucocorticoids for conditions other than VEXAS that interfere with treatment or assessment
  • More than one ICU admission due to VEXAS flare within 6 months
  • Recent high volume red blood cell transfusions within 90 days
  • Known high-risk myelodysplastic syndrome requiring treatment
  • Recent exposure to certain anti-inflammatory or hematologic treatments outside allowed timeframes
  • Use of anti-platelet therapy other than low-dose aspirin within 28 days
  • Known multiple myeloma or high serum M-protein or free light chain levels
  • Use of strong CYP3A4 inhibitors or inducers shortly before enrollment
  • Significant recent bleeding or serious cardiovascular disease or abnormal heart rhythms
  • Recent thrombotic or embolic events within 60 days
  • Moderate to severe liver impairment or active viral hepatitis
  • Uncontrolled HIV infection or positive tuberculosis test
  • History of disseminated mycobacterial infection
  • Participation in another interventional trial or recent experimental therapy
  • Pregnancy, lactation, or intention to become pregnant
  • Acute active infections requiring systemic antibiotics
  • Known allergy to pacritinib or its inactive ingredients

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Up to 24 weeks

Participants receive oral administration of pacritinib or placebo during a double-blind period to assess efficacy and safety in preventing VEXAS flares after glucocorticoid taper.

Visits scheduled throughout the 24-week double-blind treatment period

Treatment

Duration - Up to 48 weeks

Participants who complete or transition early from the double-blind period receive open-label pacritinib treatment to further assess efficacy and safety.

Visits scheduled during the open-label treatment period

Treatment

Duration - Approximately 1 year

Participants benefiting from pacritinib may continue treatment for an additional extension period of approximately 1 year to monitor long-term effects.

Visits during the extension treatment period

Follow-up

Duration - 30 days

Participants who discontinue study treatment have a 30-day follow-up period to monitor safety and health status after treatment ends.

1 follow-up visit (in-person or remote)

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

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Research Team

S

Study Physician

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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Published Research Related To This Trial

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...

https://pubmed.ncbi.nlm.nih.gov/41753113