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Tests treatment safety and results for VEXAS
Official title A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
ClinicalTrials.gov ID: NCT06782373
What this study is testing
What is Pacritinib?
Pacritinib is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for vexas.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 48 weeks
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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...
- Current or documented evidence of past inflammatory involvement within 6 months prior to enrollment of at least one of the following organ systems by...
- 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:
You likely can't join if
- 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...
- 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...
- Malignancy within 1 year prior to enrollment with the exception of MDS (per exclusion criterion), curatively treated non-melanoma skin cancer, or...
See the full eligibility criteria
Who can join
- 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...
- 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...
- 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:
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3 × upper limit of normal (ULN)
- Total bilirubin ≤4 × ULN (≤8 × ULN in the setting of Gilbert's syndrome)
- Creatinine clearance (CrCl) ≥30 mL/min based on the Cockcroft-Gault formula
- Absolute neutrophil count ≥500/μL
- Prothrombin time (PT) or international normalized ratio (INR) ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation)
- Partial thromboplastic time (PTT) or activated PTT ≤1.5 × ULN (unless prolonged due to therapeutic anticoagulation)
- Platelet count ≥25 × 10\^9/L (value must be obtained in the absence of platelet transfusion in the prior 7 days)
- 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. If QTcF is thought to be prolonged due to a...
- 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
What rules you out
- 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 how well it works.
- 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)...
- 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...
- 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:
- QT corrected by the Fridericia method (QTcF) \> 480 msec within 30 days prior to enrollment; if QTcF is thought to be prolonged due to a modifiable factor (e.g., medication / electrolyte abnormality), QTcF may be...
- Severe cardiac event (CTCAE grade ≥3) within 3 months prior to enrollment
- 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 enrollment in another treatment trial, or treatment with an 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.
The study team makes the final eligibility decision.
Where it's taking place
- Scottsdale, Arizona, United States
- Boston, Massachusetts, United States
- Rochester, Minnesota, United States
- New York, New York, United States
- Cleveland, Ohio, United States
- Columbus, Ohio, United States
- Houston, Texas, United States
- Salt Lake City, Utah, United States
- Seattle, Washington, United States
- Vancouver, British Columbia, Canada
- Halifax, Nova Scotia, Canada
- Toronto, Ontario, Canada
- Montreal, Quebec, Canada
- Lille, France
- Paris, France
- Pierre-Bénite, France
- Poitiers, France
- Toulouse, France
- Tübingen, Baden-Wurttemberg, Germany
- Munich, Bavaria, Germany
+ 17 more site(s).
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 48 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Scottsdale, Arizona, United States; Boston, Massachusetts, United States; Rochester, Minnesota, United States; New York, New York, United States; Cleveland, Ohio, United States; Columbus, Ohio, United States and 31 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.