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New treatment option for E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic...
Official title Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome
ClinicalTrials.gov ID: NCT06538181
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 e1 ubiqutin-activating enzyme, x-linked, autoinflammatory, somatic syndrome.
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
- VEXAS (vacuoles, E1 ubiqutin-activating enzyme, X-linked, autoinflammatory, somatic syndrome) is a recently described disorder with severe hematologic and rheumatologic manifestations caused by somatic variants in the ubiquitin- activating enzyme gene, UBA1, that is acquired in hematopoietic progenitor cells. Patients are often debilitated by autoinflammatory symptoms and there is currently no standard of care available.
- Phase 1: an early, usually small safety study
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
- Patients must have UBA1 mutation with a variant allele frequency (VAF) of ≥ 2% detected on a next generation sequencing panel and have at least one...
- skin rash
- vasculitis
- chondritis
- ocular/orbital inflammation (e.g., uveitis/iritis, episcleritis)
You likely can't join if
- Prior use of pacritinib.
- Use of another JAK inhibitor within 28 days of C1D1 of pacritinib.
- Currently receiving any other investigational agents. Patients may be eligible after 28 day washout.
- Thrombotic events (arterial or venous) within 60 days prior to enrollment.
- Any recent clinically significant bleeding within at least 7 days prior to enrollment.
- Any active or acute infection.
See the full eligibility criteria
- Patients must have UBA1 mutation with a variant allele frequency (VAF) of ≥ 2% detected on a next generation sequencing panel and have at least one of the following current or past clinical manifestation of VEXAS...
- skin rash
- vasculitis
- chondritis
- ocular/orbital inflammation (e.g., uveitis/iritis, episcleritis)
- genitourinary inflammation (e.g., epididymitis/orchitis)
- arthritis/arthralgias
- pulmonary inflammation (e.g., alveolitis/pleural effusion,)
- fever
- thrombosis
- splenomegaly
- hepatomegaly
- myocarditis or pericarditis
- cytopenias (defined as hemoglobin \<11 g/dL, platelets \< 100 X 10\^9 /L, OR absolute neutrophil count \<1.0 X 10\^9 /L).
- Patients with VEXAS syndrome who have never been treated with a JAK-I will be eligible to enroll on study. A stable corticosteroid dose must be maintained for at least 14 days prior to start of pacritinib.
- Patients who have previously been treated with a JAK-I other than pacritinib, or who are currently being treated with a JAK-I other than pacritinib, may be eligible after a 28 day washout if either (i) their symptoms...
- At least 18 years of age.
- ECOG performance status ≤ 3.
- Organ function as defined below:
- Absolute neutrophil count ≥ 0.5 K/cumm
- Platelets ≥ 25 K/cumm
- PT/PTT \<2.5 X upper limit of normal (ULN)
- Total bilirubin ≤ 1.5 x IULN
- AST(SGOT)/ALT(SGPT) ≤ 3.0 x IULN
- Creatinine clearance ≥ 30 mL/min by Cockcroft-Gault
- QTcF \< 480 msec.
- The effects of pacritinib on the developing human fetus are unknown. For this reason and because pacritinib was shown to be teratogenic in animal studies, women of childbearing potential and men must agree to use highly...
- Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.
- Ability to understand and willingness to sign an IRB approved written informed consent document or that of legally authorized representative, if applicable.
- Patients with myelodysplastic neoplasms (MDS) or plasma cell dyscrasias are eligible if they are not undergoing active treatment. Supportive care is permitted.
- Prior use of pacritinib.
- Use of another JAK inhibitor within 28 days of C1D1 of pacritinib.
- Currently receiving any other investigational agents. Patients may be eligible after 28 day washout.
- Thrombotic events (arterial or venous) within 60 days prior to enrollment.
- Any recent clinically significant bleeding within at least 7 days prior to enrollment.
- Any active or acute infection.
- History of malignancy within the prior 2 years, with the exception of MDS and plasma cell dyscrasias, or non-melanoma skin cancers that have been treated.
- History of clinically significant cardiovascular disease or clinically significant abnormalities in rhythm or conduction during screening EKG, including severe cardiac events, symptomatic congestive heart failure...
- Currently receiving immunosuppressants (other than corticosteroids), disease-modifying antirheumatic drugs (DMARDs), or biologic cytokine inhibitors. Patients may be eligible after 28 day washout.
- A history of allergic reactions attributed to compounds of similar chemical or biologic composition to pacritinib.
- Concurrent use of strong CYP3A4 inhibitors or inducers. Patients may be eligible after washout period of 28 days (or 5 half-lives, whichever is shorter).
- Diagnosis or history of moderate (Child-Pugh B) and severe hepatic impairment (Child-Pugh C).
- Pregnant and/or breastfeeding. Women of childbearing potential must have a negative serum pregnancy test within 7 days of C1D1 or negative urine pregnancy test within 3 days of C1D1.
- Known active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV).
- Patients with latent tuberculosis. Patients must have a negative T-Spot during screening to be eligible.
The study team makes the final eligibility decision.
Where it's taking place
- St Louis, Missouri, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
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 listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to 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 St Louis, Missouri, United States. 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.