Study of pacritinib effectiveness and safety compared to placebo in patients with VEXAS syndrome

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What is this study about?

This clinical trial examines the effectiveness and safety of pacritinib in people with VEXAS syndrome. VEXAS syndrome is a rare condition that causes inflammation in various parts of the body, including the skin, blood vessels, joints, eyes, and lungs. The condition is caused by changes in a gene called UBA1 and typically requires treatment with steroid medications.

The study will test two different doses of pacritinib capsules compared to a placebo. The medication will be taken by mouth. During the first part of the study, participants will not know whether they are receiving the actual medication or the placebo. In the second part, all participants will receive the study medication.

The study will last up to 52 weeks and will measure how well the treatment works by looking at participants’ response to the medication, including improvements in their symptoms and ability to reduce their steroid medication use. The study will also monitor changes in blood test results and track any side effects that may occur during treatment.

1 Initial visit and medication start

You will begin taking either pacritinib capsules or placebo capsules by mouth

Your current dose of prednisone or prednisolone (15-45 mg daily) will continue

Basic health checks including blood tests will be performed

2 Double-blind treatment period

You will not know whether you are receiving pacritinib or placebo

Regular monitoring of your condition and symptoms will occur

Your prednisone/prednisolone dose will be adjusted according to a fixed schedule

You will need to maintain a daily diary recording your medication use

Blood samples will be taken to monitor your health status

You will complete quality of life questionnaires about your symptoms and daily activities

3 Open-label period

After completing the double-blind period, you will enter the open-label phase

During this phase, all participants will receive pacritinib

Continued monitoring of your health status will occur

Regular blood tests and health assessments will continue

4 Health monitoring throughout study

Regular checks of your heart rhythm through ECG (heart trace) tests

Monitoring of blood counts and organ function through blood tests

Assessment of any side effects or health changes

Tracking of inflammatory markers in your blood

Regular evaluation of your response to treatment

Who Can Join the Study?

Must be 18 years or older at the time of signing the consent form
Must have confirmed genetic mutation in the UBA1 gene (specifically at position M41 or nearby) through blood or bone marrow testing
Must have current or recent (within 6 months) inflammation affecting at least one of these areas:
– Skin conditions
– Blood vessels
– Joints and cartilage
– Eyes
– Area around eyes
– Reproductive organs
– Lungs
Must be currently taking prednisone or prednisolone for at least 4 consecutive weeks
Must be on a stable dose of 15-45 mg per day of prednisone or prednisolone for at least 10 days
Must have a Karnofsky Performance Status of 50% or higher (ability to care for oneself)
Must have adequate organ function shown through specific blood tests
Must have acceptable heart rhythm measurements (QTcF)
Women who can become pregnant must have negative pregnancy tests (both blood and urine)
Both men and women must agree to use effective birth control during the study and for 30 days after
Must be willing and able to:
– Attend in-person visits
– Keep track of medication use
– Complete study procedures
– Fill out questionnaires
Must provide signed informed consent

Who Cannot Join the Study?

Prior treatment with pacritinib (the study medication)
Presence of active, severe infections requiring treatment
History of severe allergic reactions to similar medications
Significant heart problems or uncontrolled high blood pressure
Severe liver problems (as shown by blood tests)
Severe kidney problems requiring dialysis
Active cancer requiring treatment
Pregnancy or breastfeeding
Participation in other clinical trials within the past 30 days
Mental health conditions that could interfere with following study procedures
Use of medications that could interact with the study drug
Blood disorders that could affect study results
Major surgery planned during the study period
Any condition that the study doctor believes would make participation unsafe
Inability to follow study procedures or attend required visits

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Hospital Universitario De Salamanca	Salamanca	Spain
Centre Hospitalier Universitaire De Lille	Lille	France
Oncopole Claudius Regaud	Toulouse	France
Technische Universitaet Dresden	Dresden	Germany
Policlinico “Tor Vergata”, Università degli Studi di Roma TOR VERGATA	Rome	Italy

Other Sites

Site Name	City	Country
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)	Munich	Germany
Centre Hospitalier Universitaire De Poitiers	Poitiers	France
Universitaetsklinikum Schleswig-Holstein AöR	Kiel	Germany
Azienda Ospedaliera di Padova	Padua	Italy
Universitaetsklinikum Tuebingen AöR	Tuebingen	Germany
Ospedale San Raffaele S.r.l.	Milan	Italy
Hospital Clinic De Barcelona	Barcelona	Spain
Centre Hospitalier Lyon Sud	Pierre Benite	France
Hopital Beaujon	Clichy	France
Uyhpilbwib Mfdewsz Cjdrow Hngbdfzkojluiqedq	Hamburg	Germany
Iruzpazg Ckrihl Dihlecspiitwhnjgu	L'hospitalet De Llobregat	Spain
Uypwvxbvqsybollccfsbg Duqtbpizpsq Ata	Duesseldorf	Germany
Afawpnj Ufd Iwbpd Dg Rlxgbx Ekhzcf	Reggio Emilia	Italy

Trial status

Country	Status	Recruitment Start
France	Recruiting	15.05.2025
Germany	Recruiting	15.05.2025
Italy	Recruiting	15.05.2025
Spain	Recruiting	15.05.2025

Trial locations

Investigated drugs:

Pacritinib

Pacritinib is a medication being studied for treating VEXAS syndrome, a rare inflammatory condition. It works by targeting specific proteins in cells that are involved in inflammation. This medication is designed to help reduce symptoms associated with VEXAS syndrome, which can include fever, fatigue, and various inflammatory manifestations. The drug is taken orally and is being tested to determine the most effective dose for patients with this condition.

Investigated diseases:

VEXAS syndrome

VEXAS Syndrome – A rare genetic condition caused by mutations in the UBA1 gene on the X chromosome. The disease affects blood-forming cells in the bone marrow and causes inflammation throughout the body. Patients develop abnormal vacuoles (small cavities) in their blood cells, particularly in cells that fight infection. The condition typically causes recurring fevers, skin problems, and joint pain. VEXAS syndrome can also lead to fatigue and anemia, affecting blood cell production in the bone marrow. The condition primarily affects adults and causes various inflammatory symptoms that can vary in severity from person to person.

Trial ID:

2024-516347-41-00

Protocol code:

PAC601

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of pacritinib effectiveness and safety compared to placebo in patients with VEXAS syndrome

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?