Not recruitingRare disease

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

Verified siteInvestigational

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 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
Step 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
Step 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
Step 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 trial?

12 criteria

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 trial?

15 criteria

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

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Azienda Ospedaliera di Padova
Verified
Padua, Italy
Azienda USL IRCCS Di Reggio Emilia
Verified
Reggio Emilia, Italy
Centre Hospitalier Lyon Sud
Verified
Pierre Benite, France

Trial locations

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Investigated drugs

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.

Pacritinib

What is already known about the treatment

Pacritinib – An oral kinase inhibitor medication that is being investigated for the treatment of VEXAS syndrome, a rare genetic condition affecting blood cells and causing inflammation. The drug works by blocking specific enzymes called JAK2 and IRAK1, which are involved in cellular signaling and inflammation processes. Pacritinib belongs to the class of medications known as targeted therapy agents, specifically JAK inhibitors, and is administered as oral capsules. Currently, this medication is undergoing clinical trials to evaluate its safety and effectiveness in treating patients with VEXAS syndrome, with researchers studying different dose levels compared to placebo.

Investigated diseases

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.