New hope for rare inflammatory disease: pacritinib trial begins

NCT ID NCT06782373

Disease control Sponsor: Swedish Orphan Biovitrum Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Sep 16, 2026 · Updated 4 times

Summary

This study tests a drug called pacritinib for people with VEXAS syndrome, a rare genetic disease that causes severe inflammation. About 78 adults will receive either the drug or a placebo to see if it controls symptoms and reduces flare-ups. The goal is to find a safe and effective dose to manage this lifelong condition.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 156 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2025

Expected to finish

May 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Documented evidence of a pathogenic mutation at methionine-41 (M41) or neighboring splice site mutation (c.118-1, c.118-2) position in UBA1 mutation based on myeloid next-generation sequencing (NGS) droplet digital polymerase chain reaction (ddPCR), or Sanger sequencing in peripheral blood or bone marrow samples. * Current or documented evidence of past inflammatory involvement within 6 months prior to enrollment of at least one of the following organ systems by VEXAS syndrome: cutaneous (e.g., neutrophilic dermatosis, cutaneous vasculitis), vasculature (e.g., vasculitis), musculoskeletal (e.g., chondritis, arthritis), ocular (e.g., uveitis, scleritis), periorbital (e.g. periorbital edema), genitourinary (e.g., epididymitis), or pulmonary (e.g., alveolitis). * Receiving ongoing GC therapy (stable prednisone or prednisolone dose of 15-45 mg/day) leading up to enrollment. * Karnofsky Performance Status ≥50% * Adequate organ function, meeting all the following criteria within 30 days prior to enrollment: 1. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3 × upper limit of normal (ULN) 2. Total bilirubin ≤4 × ULN (≤8 × ULN in the setting of Gilbert's syndrome) 3. Creatinine clearance (CrCl) ≥30 mL/min based on the Cockcroft-Gault formula 4. Absolute neutrophil count ≥500/μL 5. Prothrombin time (PT) or international normalized ratio (INR) ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation) 6. Partial thromboplastic time (PTT) or activated PTT ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation) 7. Platelet count ≥25 × 10\^9/L (value must be obtained in the absence of platelet transfusion in the prior 7 days) 8. Peripheral blasts \<5% * QT corrected by the Fridericia method (QTcF) ≤450 msec in males or ≤470 msec in females. Participants with QRS prolongation \>100 msec may enroll if their QTcF is ≤480 msec. Participants with ventricular paced rhythms may enroll if their QTcF is ≤500 msec. If QTcF is thought to be prolonged due to a modifiable factor (e.g., medication / electrolyte abnormality), QTcF may be reevaluated. * Women of child-bearing potential (WOCBP) must have a negative serum pregnancy test within 30 days prior to enrollment and a negative urine pregnancy test on Day 1 prior to randomization and dosing. * WOCBP and male participants must agree to use a highly effective method of contraception starting at the first dose of trial therapy through 30 days after the last dose of trial therapy. Key Exclusion Criteria * Prior allogenic hematopoietic stem cell transplant (allo-HSCT) or solid organ transplant (other than corneal). * Current use of systemic GCs for conditions other than VEXAS syndrome, which, in the opinion of the Investigator, would interfere with adherence to a GC taper regimen and/or assessment of efficacy. * More than one prior admission to an intensive care unit due to a VEXAS Syndrome flare within the prior 6 months. * Received ≥9 units of intensive red blood cell (RBC) transfusions in the 90 days prior to enrollment. * Known concurrent myelodysplastic syndrome (MDS) requiring antineoplastic treatment, or allo-HSCT, or known high-risk or very high-risk MDS based on the Revised International Prognostic Scoring System (IPSS-R). Participants with MDS who do not meet these criteria may enroll. * Malignancy within 1 year prior to enrollment with the exception of MDS (per exclusion criterion), curatively treated non-melanoma skin cancer, or curatively treated carcinoma in situ. Participants with pre-malignant hematologic conditions (e.g., monoclonal gammopathy of unknown significance \[MGUS\], clonal cytopenia of unknown significance) may enroll. * Exposure to hypomethylating agents (HMA) within 6 months prior to enrollment, or exposure to more than 6 cycles of HMAs at any time. * Exposure to non-GC anti-inflammatory therapy or hematologic support therapy within protocol defined timeframes prior to enrollment * Exposure to anti-platelet therapy with the exception of low-dose aspirin (≤100 mg daily) within 28 days prior to enrollment. * Known concomitant multiple myeloma, or serum M-protein ≥3 g/dL, involved-to uninvolved free light chain (FLC) ratio ≥100, or involved FLC level ≥100 mg/dL. Participants with MGUS may enroll. * Systemic treatment with a strong cytochrome P450 3A4 (CYP3A4) inhibitor or inducer within 5 half-lives prior to enrollment. * Significant recent bleeding history defined as National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) grade ≥2 within 3 months prior to enrollment, unless precipitated by an inciting event. * History of clinically significant cardiovascular disease, or clinically significant abnormalities in rhythm or conduction during Screening ECG, including: 1. Severe cardiac event (CTCAE grade ≥3) within 3 months prior to enrollment 2. Heart failure resulting in limitations during ordinary activity. * Arterial or venous thrombotic or embolic events, including deep vein thrombosis, pulmonary embolism, and cerebrovascular accident (including transient ischemic attacks), within 60 days prior to enrollment. * Moderate or severe hepatic impairment that meets criteria for Child-Pugh Class B or C, or active viral hepatitis. * Uncontrolled human immunodeficiency virus (HIV) off antiretrovirals, or on antiretrovirals with detectable viral load. * Positive Quantiferon (or other interferon gamma release assay) during Screening. * Known history of disseminated mycobacterial infection. * Concurrent or prior enrollment in another prospective interventional trial of VEXAS-directed therapy, or concurrent enrollment in another interventional trial of non-VEXAS-directed therapy, or treatment with a non-VEXAS-directed experimental therapy within 28 days or five half-lives prior to enrollment, whichever is longer. * Pregnant, intending to become pregnant during the trial, or currently breastfeeding/lactating. * Participants with any acute, active infection requiring systemic antimicrobial treatment at the time of enrollment. Exceptions are made for prophylactic antibiotics or chronic antibiotic therapy for non-acute conditions. * Known hypersensitivity to pacritinib or any of the following inactive ingredients: microcrystalline cellulose, polyethylene glycol, and magnesium stearate.

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Conditions

The condition(s) this trial relates to.

hematopoietic and lymphoid system neoplasm Myelodysplastic Syndromes VEXAS syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

VEXAS Vexas syndrome

Contacts and locations

Locations

AUSL of Reggio Emilia - Hospital Arcispedale S. Maria Nuova, Complex Structure of Rheumatology

Reggio Emilia, 42123, Italy
Catalan Institute of Oncology, Hospital Duran i Reynals, Department of Clinical Hematology

L'Hospitalet de Llobregat, 08908, Spain
Churchill Hospital

Oxford, OX3 7LE, United Kingdom
Cleveland Clinic - Cleveland

Cleveland, Ohio, 44195, United States
Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Foundation PTV - Polyclinic Tor Vergata Biomedicine and prevention

Roma, 00133, Italy
Fred Hutchinson Cancer Center

Seattle, Washington, 98109, United States
Fukushima Medical University Hospital

Fukushima, 960-1295, Japan
Hospices Civils de Lyon - Lyon Sud

Pierre-Bénite, 69310, France
Hospital Clinic of Barcelona

Barcelona, 08036, Spain
Hospital Rechts der Isar of the Technical University of Munich, Clinic and Polyclinic for Internal Medicine III: Hematology and Internal Oncology

Munich, Bavaria, 81675, Germany
Hospital San Raffaele, IRCCS, Unit of Immunology, Rheumatology, Allergy and Rare Diseases

Milan, 20132, Italy
Hospital du Sacre-Coeur in Montreal

Montreal, Quebec, H4J 1C5, Canada
IUCT-Oncopole

Toulouse, 31100, France
King's College Hospital, Department of Hematology

London, SE5 9RS, United Kingdom
Lille University Hospital Center

Lille, 59037, France
Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States
Mayo Clinic - Scottsdale

Scottsdale, Arizona, 85259, United States
NYU Langone Health

New York, New York, 10016, United States
Nagasaki University Hospital

Nagasaki, 852-8501, Japan
Princess Margaret Cancer Centre

Toronto, Ontario, M5G 2M9, Canada
Queen Elizabeth II Health Sciences Center

Halifax, Nova Scotia, B3H 2Y9, Canada
Royal Free Hospital

London, NW3 2QG, United Kingdom
Saint-Antoine Hospital - APHP

Paris, 75012, France
St James's University Hospital

Leeds, LS9 7TF, United Kingdom
Tenon Hospital - APHP

Paris, 75020, France
The James Cancer Hospital and Solove Research Institute

Columbus, Ohio, 43210, United States
UT MD Anderson Cancer Center

Houston, Texas, 77030, United States
University Clinical Hospital of Salamanca

Salamanca, 37007, Spain
University Hospital Carl Gustav Carus Dresden, Medical Clinic and Polyclinic I

Dresden, Saxony, 01307, Germany
University Hospital Center of Poitiers

Poitiers, 86000, France
University Hospital Duesseldorf

Düsseldorf, North Rhine-Westphalia, 40225, Germany
University Hospital Hamburg-Eppendorf

Hamburg, Free and Hanseatic City of Hamburg, 20246, Germany
University Hospital Schleswig-Holstein

Lübeck, Schleswig-Holstein, 23538, Germany
University Hospital Tuebingen, Medical Clinic II, Hematology, Oncology, Clinical Immunology and Rheumatology

Tübingen, Baden-Wurttemberg, 72076, Germany
University Hospital of Padova, Rheumatology Unit, Department of Medicine - DIMED

Padova, 35128, Italy
University of Utah Healthcare

Salt Lake City, Utah, 84132, United States
Vancouver Coastal Health Research Institute

Vancouver, British Columbia, V5Z 1M9, Canada
Yokohama City University Hospital

Yokohama, 236-0004, Japan

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