RecruitingPhase 2NCT06782373

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

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

Sponsor

Swedish Orphan Biovitrum

Enrollment

78 participants

Start Date

May 28, 2025

Study Type

INTERVENTIONAL

Conditions

VEXAS Syndrome VEXAS

Summary

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.

Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Pacritinib and a drug called Placebo for people with vexas and vexas syndrome. The study is currently recruiting participants at 39 locations. People eligible for this study include aged 18 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGPacritinib

Supplied in hard capsules.

DRUGPlacebo

Supplied in hard capsules.

Locations(39)

Mayo Clinic - Scottsdale

Scottsdale, Arizona, United States

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Mayo Clinic - Rochester

Rochester, Minnesota, United States

NYU Langone Health

New York, New York, United States

Cleveland Clinic - Cleveland

Cleveland, Ohio, United States

The James Cancer Hospital and Solove Research Institute

Columbus, Ohio, United States

UT MD Anderson Cancer Center

Houston, Texas, United States

University of Utah Healthcare

Salt Lake City, Utah, United States

Fred Hutchinson Cancer Center

Seattle, Washington, United States

Vancouver Coastal Health Research Institute

Vancouver, British Columbia, Canada

Queen Elizabeth II Health Sciences Center

Halifax, Nova Scotia, Canada

Princess Margaret Cancer Centre

Toronto, Ontario, Canada

Hospital du Sacre-Coeur in Montreal

Montreal, Quebec, Canada

Lille University Hospital Center

Lille, France

Saint-Antoine Hospital - APHP

Paris, France

Tenon Hospital - APHP

Paris, France

Hospices Civils de Lyon - Lyon Sud

Pierre-Bénite, France

University Hospital Center of Poitiers

Poitiers, France

IUCT-Oncopole

Toulouse, France

University Hospital Tuebingen, Medical Clinic II, Hematology, Oncology, Clinical Immunology and Rheumatology

Tübingen, Baden-Wurttemberg, Germany

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

Munich, Bavaria, Germany

University Hospital Hamburg-Eppendorf

Hamburg, Free and Hanseatic City of Hamburg, Germany

University Hospital Duesseldorf

Düsseldorf, North Rhine-Westphalia, Germany

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

Dresden, Saxony, Germany

University Hospital Schleswig-Holstein

Lübeck, Schleswig-Holstein, Germany

Hospital San Raffaele, IRCCS, Unit of Immunology, Rheumatology, Allergy and Rare Diseases

Milan, Italy

University Hospital of Padova, Rheumatology Unit, Department of Medicine - DIMED

Padova, Italy

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

Reggio Emilia, Italy

Foundation PTV - Polyclinic Tor Vergata Biomedicine and prevention

Roma, Italy

Fukushima Medical University Hospital

Fukushima, Japan

Nagasaki University Hospital

Nagasaki, Japan

Yokohama City University Hospital

Yokohama, Japan

Hospital Clinic of Barcelona

Barcelona, Spain

Catalan Institute of Oncology, Hospital Duran i Reynals, Department of Clinical Hematology

L'Hospitalet de Llobregat, Spain

University Clinical Hospital of Salamanca

Salamanca, Spain

St James's University Hospital

Leeds, United Kingdom

Royal Free Hospital

London, United Kingdom

King's College Hospital, Department of Hematology

London, United Kingdom

Churchill Hospital

Oxford, United Kingdom

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NCT06782373

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